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Coronavirus (COVID-19) Update: Daily Roundup June 22, 2020

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Coronavirus (COVID-19) Update: Daily Roundup June 22, 2020
June 22, 2020: “The U.S. Food and Drug Administration announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:
  • The FDA issued a guidance document, titled “Effects of the COVID-19 Public Health Emergency on Formal Meetings and User Fee Applications for Medical Devices – Questions and Answers,” with answers to frequently asked questions.

    These include answers concerning certain aspects of sponsor requests for formal meetings with the FDA, user-fee application goals and timelines, and other regulatory and policy issues related to device development for the duration of the COVID-19 public health emergency.
  • As part of the FDA’s continuing effort to protect consumers, the agency issued a warning letter to one firm for selling fraudulent COVID-19 products. The seller, North Isle Wellness Center, offers Methylene Blue products for sale in the United States with misleading claims that the products can mitigate, prevent, treat, , or cure COVID-19 in people.

    The letter requests that the seller take immediate action to cease the sale of such unapproved and unauthorized products.

    There are currently no FDA-approved products to prevent or treat COVID-19. Consumers concerned about COVID-19 should consult with their health care provider.
  • Testing updates:
    • To date, the FDA has authorized 145 tests under EUAs; these include 122 molecular tests, 22 antibody tests, and 1 antigen test.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-daily-roundup-june-22-2020

FDA Accepts sBLA for BOTOX® (onabotulinumtoxinA) for the Treatment of Pediatric Patients with Neurogenic Detrusor Overactivity

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FDA Accepts sBLA for BOTOX® (onabotulinumtoxinA) for the Treatment of Pediatric Patients with Neurogenic Detrusor Overactivity
 June 22, 2020: “Allergan an AbbVie company announced that the U.S.FDA has accepted the company’s supplemental biologics license application (sBLA) to expand the BOTOX® prescribing information for the treatment of signs and symptoms of detrusor (bladder muscle) overactivity associated with an underlying neurologic condition (e.g., spina bifida, spinal cord injuries) in pediatric patients (5 -17 years of age) who have an inadequate response to, or are intolerant of, or for any reason unwilling to continue anticholinergic medication.

The sBLA is based on data from a randomized, double-blind Phase 3 study evaluating the safety and efficacy of BOTOX® in more than 100 pediatric patients with neurogenic detrusor overactivity and a long-term extension study.

The Prescription Drug User Fee Act date is expected to be in the first quarter of 2021 following a standard 10-month review.

“The FDA acceptance of this application underscores our ongoing commitment to pursuing the full potential of BOTOX® to serve patients across a wide spectrum of diseases and clinical needs,” said Mitchell F. Brin, M.D., Senior Vice President, Chief Scientific Officer, BOTOX® & Neurotoxins, AbbVie.

“Children living with neurogenic detrusor overactivity currently have limited options when they fail anticholinergic medications and prior to surgical intervention.

If approved, BOTOX® will be the first neurotoxin treatment approved for use in treating detrusor overactivity in pediatric patients with an underlying neurologic condition who are not adequately managed by anticholinergic medications.”

Neurogenic detrusor overactivity results from the inability of the spinal cord and bladder to communicate effectively.

As a result, the bladder muscle (detrusor) involuntarily contracts, increasing the pressure in the bladder and reducing the bladder capacity, which can cause the individual to leak urine frequently and unexpectedly.

If not adequately managed with clean intermittent catheterization and anticholinergic drugs, this condition may require augmentation cystoplasty (an extensive surgical procedure in which the bladder is enlarged using a section of the patient’s intestine) to prevent renal damage.

There are a number of causes of neurogenic detrusor overactivity in children, such as transverse myelitis, spinal cord injury, and spina bifida, which is the most common and affects 1,500-2,000 of the more than 4 million babies born in the United States each year. 

More than 90% of those living with spina bifida experience urinary symptoms.

“Over time, many pediatric patients with underlying neurologic conditions experience bladder and kidney damage, and treatment is critical,” said Paul F. Austin, M.D., FAAP, Chief of Pediatric Urology at Texas Children’s Hospital and Professor of Urology at Baylor College of Medicine.

“Current treatment options often include anticholinergic medications, where long-term use needs to be considered carefully, in addition to surgery. The favorable BOTOX® clinical results for the treatment of pediatric patients with neurogenic detrusor activity are promising as we look to address unmet and ongoing needs in children and adolescents.”

BOTOX® is the first and only neurotoxin approved for the treatment of leakage of urine (incontinence) due to overactive bladder caused by a neurologic condition in adults who still have leakage or cannot tolerate the side effects after trying an anticholinergic medication.

BOTOX® temporarily reduces muscle contractions by blocking the transmission of nerve impulses to the muscle, in this case, the bladder muscle, by selectively preventing the release of the neurotransmitter acetylcholine at the neuromuscular junction.

BOTOX®
BOTOX® is one of the most widely researched medications in the world, with a proven history as a therapeutic agent.

First approved by the FDA in 1989 for two rare eye muscle disorders – blepharospasm and strabismus in adults, BOTOX® was the world’s first approved botulinum toxin type A treatment.

Today, BOTOX® is FDA-approved for 11 therapeutic indications, including Chronic Migraine, overactive bladder, leakage of urine (incontinence) due to overactive bladder caused by a neurologic condition, cervical dystonia, spasticity, and severe underarm sweating (axillary hyperhidrosis).

Backed by strong science and continuous innovation, BOTOX® proudly embraces its past while boldly looking to the future.

BOTOX® (onabotulinumtoxinA) Important Information

Indications
BOTOX® is a prescription medicine that is injected into muscles and used:

  • To treat overactive bladder symptoms such as a strong need to urinate with leaking or wetting accidents (urge urinary incontinence), a strong need to urinate right away (urgency), and urinating often (frequency) in adults 18 years and older when another type of medicine (anticholinergic) does not work well enough or cannot be taken
  • To treat leakage of urine (incontinence) in adults 18 years and older with overactive bladder caused by a neurologic disease who still have leakage or cannot tolerate the side effects after trying an anticholinergic medication
  • To prevent headaches in adults with Chronic Migraine who have 15 or more days each month with headache lasting 4 or more hours each day in people 18 years or older
  • To treat increased muscle stiffness in elbow, wrist, finger, and thumb muscles in people 18 years and older with upper limb spasticity
  • To treat increased muscle stiffness in ankle and toe muscles in people 18 years and older with lower limb spasticity
  • To treat increased muscle stiffness in children 2 to 17 years of age with upper limb spasticity
  • To treat increased muscle stiffness in children 2 to 17 years of age with lower limb spasticity, excluding spasticity caused by cerebral palsy
  • To treat the abnormal head position and neck pain that happens with Cervical Dystonia (CD) in people 16 years and older
  • To treat certain types of eye muscle problems (Strabismus) or abnormal spasm of the eyelids (Blepharospasm) in people 12 years and older

BOTOX® is also injected into the skin to treat the symptoms of severe underarm sweating (severe primary axillary hyperhidrosis) when medicines used on the skin (topical) do not work well enough in people 18 years and older.

It is not known whether BOTOX® is safe or effective to prevent headaches in patients with migraine who have 14 or fewer headache days each month (episodic migraine).

It is not known whether BOTOX® is safe or effective to treat increased stiffness in upper limb muscles other than those in the elbow, wrist, fingers, and thumb, or in lower limb muscles other than those in the ankle and toes in people 18 years and older.

BOTOX® has not been shown to help people perform task-specific functions with their upper limbs or increase movement in joints that are permanently fixed in position by stiff muscles.

It is not known whether BOTOX® is safe or effective for other types of muscle spasms or for severe sweating anywhere other than your armpits.

IMPORTANT SAFETY INFORMATION

BOTOX® may cause serious side effects that can be life threatening. Get medical help right away if you have any of these problems any time (hours to weeks) after injection of BOTOX®:

  • Problems swallowing, speaking, or breathing, due to weakening of associated muscles, can be severe and result in loss of life. You are at the highest risk if these problems are pre-existing before injection. Swallowing problems may last for several months
     
  • Spread of toxin effects. The effect of botulinum toxin may affect areas away from the injection site and cause serious symptoms including: loss of strength and all-over muscle weakness, double vision, blurred vision and drooping eyelids, hoarseness or change or loss of voice, trouble saying words clearly, loss of bladder control, trouble breathing, and trouble swallowing

There has not been a confirmed serious case of spread of toxin effect away from the injection site when BOTOX® has been used at the recommended dose to treat Chronic Migraine, severe underarm sweating, Blepharospasm, or Strabismus.

BOTOX® may cause loss of strength or general muscle weakness, vision problems, or dizziness within hours to weeks of taking BOTOX®. If this happens, do not drive a car, operate machinery, or do other dangerous activities.“
https://news.abbvie.com/news/press-releases/fda-accepts-supplemental-biologics-license-application-sbla-for-botox-onabotulinumtoxina-for-treatment-pediatric-patients-with-neurogenic-detrusor-overactivity.htm

Cipla launches Cipremi (remdesivir lyophilised powder for injection 100 mg), the only U.S. FDA approved EUA treatment for patients with severe COVID-19 disease

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Cipla launches Cipremi (remdesivir lyophilised powder for injection 100 mg), the only U.S. FDA approved EUA treatment for patients with severe COVID-19 disease

June 21, 2020: “Cipla Limited announced the launch of remdesivir under its brand name CIPREMI.

The U.S. FDA issued an Emergency Use Authorization (EUA) to Gilead Sciences Inc. for emergency use of remdesivir for the treatment of hospitalized 2019 coronavirus disease (COVID-19) patients.

It is the only U.S. FDA approved Emergency Use Authorisation (EUA) treatment for adult and paediatric patients hospitalized with suspected or laboratory confirmed COVID-19 infection.

In May, Gilead Sciences Inc. extended a voluntary non-exclusive license to Cipla to manufacture and market Cipla’s generic version of remedisvir called CIPREMI.

Cipla has been granted regulatory approval by the Drug Controller General of India (DCGI) for restricted emergency use in the country as part of the accelerated approval process considering the urgent and unmet medical need.

As part of a risk management plan, Cipla will provide training on use of the drug, informed patient consent documents, conduct post marketing surveillance as well as conduct a Phase IV clinical trial on Indian patients.

According to a preliminary report from the ACTT-1 (Adaptive COVID-19 Treatment Trial 1) study1 , a randomized clinical trial conducted with remdesivir in 1063 patients over 60 centres across U.S., Europe and Asia demonstrated a faster time to clinical recovery in hospitalised patients as compared to placebo.

Most of these patients were on oxygen therapy of which some were receiving high flow oxygen or non-invasive ventilation, and some were on a mechanical ventilator.

The mortality rates in the study were 7.1% in those given remdesivir and 11.9% in those who were given placebo.

As part of its efforts to enable speedy and equitable access to this treatment and in anticipation of demand, Cipla will be commercialising remdesivir through its own facilities and partnered sites. The drug will be supplied through Government and open market channels, to ensure equitable distribution.

Commenting on the launch, Mr. Umang Vohra (MD and Global CEO, Cipla Limited) said, “Cipla appreciates the strong partnership with Gilead to bring remdesivir to patients in India.

We have been deeply invested in exploring all possible avenues to save millions of lives impacted by COVID-19 pandemic, and this launch is a significant milestone in that direction. “

We will continue to collaborate with all stakeholders in the healthcare ecosystem towards providing access to such promising treatments in furtherance with our belief that no patient should be denied access to life-saving treatments.”
https://www.cipla.com/press-releases-statements/cipla-launches-cipremi-remdesivir-lyophilised-powder-injection-100-mg

FDA Oks Crysvita® for the Treatment of Tumor-Induced Osteomalacia (TIO)

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FDA Oks Crysvita® for the Treatment of Tumor-Induced Osteomalacia (TIO)
 June 18, 2020: “Ultragenyx Pharmaceutical announced that the U.S. FDA has approved Crysvita® (burosumab) for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.

Crysvita is a human antibody that blocks excess activity of FGF23, a hormone that causes phosphate urinary excretion and suppresses active vitamin D production by the kidney.

“For approximately half of all individuals with TIO, surgical removal of the tumors is not possible, leaving these patients with no other treatment options.

The FDA approval of Crysvita marks the first treatment option that addresses the cause of the severe hypophosphatemia and osteomalacia resulting from these rare tumors,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

“We plan to leverage our experience and existing infrastructure with Crysvita in X-linked hypophosphatemia to bring this important medicine to patients living with the rare, painful and debilitating disorder of TIO.”

“Since its approval, Crysvita has meant a great deal to patients and families that previously had no other treatment options.

We are proud of the work that has been done to advance this discovery from our labs, through a robust clinical research program, and through the FDA’s priority review process, to make this treatment available to patients with TIO,” said Gary Zieziula, President, North America for Kyowa Kirin.

“Our commitment to meeting the needs of patients with rare and serious diseases remains steadfast and we will continue to partner with the Ultragenyx team to address these needs with urgency.”

TIO is a rare disease caused by typically benign, slow-growing tumors that produce excess levels of FGF23, which is involved in phosphate reabsorption. Patients with TIO can experience symptoms including severe hypophosphatemia (low levels of phosphate in the blood), osteomalacia (softening of the bones), muscle weakness, fatigue, bone pain and fractures

There are an estimated 500 to 1,000 people in the United States with TIO, and approximately half of all cases are believed to be inoperable.

In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or active vitamin D replacement. 

Efficacy of this management is often limited, and its benefits must be balanced with monitoring for potential risks.

This is the second FDA-approved indication for Crysvita, which was first approved in April 2018 for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older.

The XLH indication was expanded in September 2019 to include infants as young as six months of age.

The FDA approval of Crysvita for TIO was based on data from two single-arm Phase 2 studies, a 144-week study in 14 adult patients conducted by Ultragenyx in the United States and an 88-week study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels.

Related News: Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) for the treatment of Tumor-Induced Osteomalacia (TIO)

FDA Approves First Therapy for Rare Disease

Increased phosphate levels led to improvements in osteomalacia. Additionally, whole body bone scans demonstrated reduced tracer uptake with long-term treatment suggesting healing of bone lesions. Most common adverse reactions (>10%) in TIO patients are: tooth abscess, muscle spasms, dizziness, constipation, injection site reaction, rash, and headache.

The FDA granted Priority Review designation for the supplemental BLA for TIO, which is reserved for drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness.

To support access, Ultragenyx has a program called UltraCare™, which helps patients and caregivers understand insurance coverage and assists them in finding financial support for Ultragenyx medicines including Crysvita, and for the administration of them.

Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657.

Crysvita
Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney.

Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23.

Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium.

Crysvita is approved by the U.S. FDA for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older, and by Health Canada and Brazil’s National Health Surveillance Agency (ANVISA) for the treatment of XLH in adult and pediatric patients one year of age and older.

In Japan, it is approved by the Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia.

In Europe, Crysvita has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency. 

Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

U.S. INDICATION
Crysvita® (burosumab-twza) is a fibroblast growth factor 23 (FGF23)-blocking antibody indicated for the treatment of:

  • X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.
  • FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS

  • With oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol).
  • When serum phosphorus is within or above the normal range for age.
  • In patients with severe renal impairment or end stage renal disease.        

WARNINGS AND PRECAUTIONS
Hypersensitivity

  • Discontinue Crysvita if serious hypersensitivity reactions occur and initiate appropriate medical treatment.           

Hyperphosphatemia and Risk of Nephrocalcinosis

  • For patients already taking Crysvita, dose interruption and/or dose reduction may be required based on a patient’s serum phosphorus levels.
  • Patients with TIO who undergo treatment of the underlying tumor should have dosing interrupted and adjusted to prevent hyperphosphatemia.

Injection Site Reactions

  • Discontinue Crysvita if severe injection site reactions occur and administer appropriate medical treatment.

ADVERSE REACTIONS
Pediatric XLH Patients

  • Adverse reactions reported in 10% or more of Crysvita-treated pediatric XLH patients across all studies are: pyrexia, injection site reaction, cough, vomiting, pain in extremity, headache, tooth abscess, dental caries, diarrhea, vitamin D decreased, toothache, constipation, myalgia, rash, dizziness, and nausea.
  • Post-marketing experience reported in pediatric XLH patients receiving Crysvita – blood phosphorus increased.

             
Adult XLH Patients

  • Adverse reactions reported in more than 5% of Crysvita-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain, headache, tooth infection, restless legs syndrome, vitamin D decreased, dizziness, constipation, muscle spasms, and blood phosphorus increased.
  • Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if Crysvita therapy exacerbates spinal stenosis or spinal cord compression.

Adult TIO Patients

  • Adverse reactions reported in more than 10% of Crysvita-treated adult TIO patients in two studies are: tooth abscess, muscle spasms, dizziness, constipation, injection site reaction, rash, and headache.

USE IN SPECIFIC POPULATIONS

  • There are no available data on Crysvita use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at
    1-888-756-8657.
  • There is no information regarding the presence of Crysvita in human milk or the effects of Crysvita on milk production or the breastfed infant.

PATIENT COUNSELING INFORMATION

FDA Approves Evoke’s GIMOTI™

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FDA Approves Evoke’s GIMOTI™

June 19, 2020: Evoke Pharma announced that the U.S. FDA has approved the New Drug Application (NDA) for GIMOTI™ (metoclopramide) nasal spray, the first and only nasally-administered product indicated for the relief of symptoms in adults with acute and recurrent diabetic gastroparesis.

“We are extremely pleased to have received FDA approval to commercially market GIMOTI in the United States.

This approval represents the first novel pharmaceutical treatment for gastroparesis in several decades.

Many times, patients do not experience adequate relief of their gastroparesis symptoms from current treatments, representing a significant need for a new approach to therapy. We are excited to be able to offer health care providers and their patients a unique non-oral treatment option to relieve symptoms and help improve their quality of life,” said David Gonyer, R.Ph., President and CEO.

“Patients with gastroparesis suffer from characteristic symptoms such as nausea, abdominal pain, bloating, early satiety as well as vomiting which can be severe and debilitating.

These patients often have erratic absorption of orally administered drugs due to delayed gastric emptying,” stated Henry Parkman, MD, Stanley H. Lorber Research Endowment Fund and Chair, and Director, Gastroenterology Motility Laboratory, School of Medicine at Temple University.

“Unlike oral medications, GIMOTI is administered nasally, bypassing the diseased GI track, allowing the drug to enter the bloodstream directly and therefore may provide predictable delivery of the therapy.”

“Together with our partner EVERSANA, we are now fully focused on executing our commercialization strategy for GIMOTI by leveraging EVERSANA’s integrated suite of capabilities and highly experienced sales and marketing team,” continued David Gonyer, R.Ph.

“We anticipate initiating commercial sales in the fourth quarter of 2020.” 

The FDA approval of GIMOTI allows Evoke to access its existing $5 million line of credit from EVERSANA to support manufacturing and other aspects of GIMOTI’s commercialization.

As of May 31, 2020, the Company’s cash and cash equivalents were approximately $4.7 million.

Evoke believes, based on its current operating plan, that its cash and cash equivalents, together with the EVERSANA line of credit, will support the company’s operations into 2021, without consideration of potential GIMOTI revenue.

GIMOTI™ (metoclopramide) nasal spray
GIMOTI is indicated for the relief of symptoms in adults with acute and recurrent diabetic gastroparesis.

Important Safety Information
WARNING: TARDIVE DYSKINESIA

  • Metoclopramide can cause tardive dyskinesia (TD), a serious movement disorder that is often irreversible. The risk of developing TD increases with duration of treatment and total cumulative dosage.
  • Discontinue GIMOTI in patients who develop signs or symptoms of TD. In some patients, symptoms may lessen or resolve after metoclopramide is stopped.
  • Avoid treatment with metoclopramide (all dosage forms and routes of administration) for longer than 12 weeks because of the increased risk of developing TD with longer-term use.

GIMOTI is not recommended for use in:

  • Pediatric patients due to the risk of developing tardive dyskinesia (TD) and other extrapyramidal symptoms as well as the risk of methemoglobinemia in neonates.
  • Moderate or severe hepatic impairment (Child‑Pugh B or C), moderate or severe renal impairment (creatinine clearance less than 60 mL/minute), and patients concurrently using strong CYP2D6 inhibitors due to the risk of increased drug exposure and adverse reactions.

GIMOTI is contraindicated:

  • In patients with a history of tardive dyskinesia (TD) or a dystonic reaction to metoclopramide.
  • When stimulation of gastrointestinal motility might be dangerous (e.g., in the presence of gastrointestinal hemorrhage, mechanical obstruction, or perforation).
  • In patients with pheochromocytoma or other catecholamine-releasing paragangliomas. Metoclopramide may cause a hypertensive/pheochromocytoma crisis, probably due to release of catecholamines from the tumor.
  • In patients with epilepsy. Metoclopramide may increase the frequency and severity of seizures.
  • In patients with hypersensitivity to metoclopramide. Reactions have included laryngeal and glossal angioedema and bronchospasm.

Potential adverse reactions associated with metoclopramide include:  Tardive dyskinesia (TD), other extrapyramidal effects (EPS), parkinsonism symptoms, motor restlessness, neuroleptic malignant syndrome (NMS), depression, suicidal ideation and suicide, hypertension, fluid retention, hyperprolactinemia, effects on the ability to drive and operate machinery.

Most common adverse reactions (≥5%) for GIMOTI are: dysgeusia, headache, and fatigue. 

These are not all of the possible side effects of GIMOTI. Call your doctor for medical advice about whether you should take GIMOTI and the possible risk factors and side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Evoke Pharma, Inc.
Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases.

The Company developed GIMOTI, a nasal spray formulation of metoclopramide, for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adults.

Diabetic gastroparesis is a GI disorder affecting millions of patients worldwide, in which the stomach takes too long to empty its contents resulting in serious GI symptoms as well as other systemic complications. The gastric delay caused by gastroparesis can compromise absorption of orally administered medications.

Prior to FDA approval to commercially market GIMOTI, metoclopramide was only available in oral and injectable formulations and remains the only drug currently approved in the United States to treat gastroparesis. Visit www.EvokePharma.com for more information.
https://fda.einnews.com/pr_news/519857656/fda-approves-evoke-s-gimoti?ref=rss&ecode=YpDfNa50_yJ5LLJS&utm_source=RSSNews&utm_medium=rss&utm_campaign=FDA+News&utm_content=article

Dupixent® approved in China for adults with atopic dermatitis

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Dupixent® approved in China for adults with atopic dermatitis

June 19, 2020: The National Medical Products Administration (NMPA) in China has approved Dupixent® (dupilumab) for the treatment of moderate-to-severe atopic dermatitis in adults whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

The NMPA identified Dupixent as an overseas medicine considered urgently needed in clinical practice, leading to an expedited review and approval process.

“The limited treatment options in China for moderate-to-severe atopic dermatitis has left many patients and those who care for them coping with the physical and emotional burden of the disease,” said Professor Zhang Jianzhong, chairman of the 13th session of the Dermatology and Venereology Branch of the Chinese Medical Association, and director of the Department of Dermatology, Peking University People’s Hospital. 

“The availability of a targeted treatment like Dupixent provides hope to those seeking relief from the often-unbearable itch and other symptoms that can significantly impact the lives of adults living with this chronic disease.”

Atopic dermatitis is a chronic inflammatory disease that often appears as a rash on the skin.

Moderate-to-severe atopic dermatitis is characterized by rashes that can potentially cover much of the body, and can include intense, persistent itching, skin dryness and skin lesions including cracking, redness or darkness, crusting and oozing.

Itch is one of the most burdensome symptoms for patients and can be debilitating.

Inadequately controlled atopic dermatitis can have a physical, emotional and psychosocial impact, causing sleep disturbance, symptoms of anxiety and depression, and feelings of isolation.

“Sanofi has deep roots in China, and it continues to be a significant area of growth for us. New regulations have paved the way for first-in-class treatments like Dupixent to be delivered to patients sooner and, in partnership with the government’s Healthy China 2030 initiative, we plan to seek approval by 2025 for more than 25 innovative medicines for chronic and rare diseases and vaccines,” said Paul Hudson, Chief Executive Officer, Sanofi. 

“The approval of Dupixent in China offers a new treatment option with an established safety and efficacy profile. This is a meaningful advance for patients and their physicians who have struggled to treat the debilitating symptoms of moderate-to-severe atopic dermatitis which can seriously impact quality of life.”

Dupixent is a fully-human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) proteins, and is not an immunosuppressant.

Data from Dupixent clinical trials have shown that IL-4 and IL-13 are key drivers of the type 2 inflammation that plays a major role in atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP).

“As the first biologic medicine approved for moderate-to-severe atopic dermatitis, Dupixent has transformed the treatment landscape for patients around the world by targeting the type 2 inflammation that underlies the disease, rather than broadly suppressing the immune system,” said George D. Yancopoulos, M.D., Ph.D., Co-founder, President and Chief Scientific Officer of Regeneron. 

“More than 150,000 people have already been treated with Dupixent globally and today’s approval brings this novel treatment to those in China who are in urgent need of new options.”

The approval was based on positive data from the global LIBERTY AD clinical trial program that included nearly 3,000 patients with inadequately controlled moderate-to-severe atopic dermatitis.

The trials evaluated Dupixent (monotherapy or in combination with topical corticosteroids) on safety and efficacy measures, including skin clearance, overall disease severity and itch.

Data from an ongoing Phase 3 trial in China of adults with moderate-to-severe atopic dermatitis will be shared with NMPA in the second half of 2020 when the trial has completed.

Dupixent
Dupixent will be available in China in a 300 mg dose as a pre-filled syringe. Dupixent is intended for injection under the skin (subcutaneous injection) and is given every other week following an initial loading dose.

It can be given in a clinic or at home by self-administration after training by a healthcare professional. Dupixent can be used with or without topical corticosteroids.

Dupixent is currently approved in the U.S., Europe, Japan and other countries around the world for use in specific patients with moderate-to-severe atopic dermatitis, as well as certain patients with asthma or CRSwNP in different age populations.

Dupilumab Development Program
To date, dupilumab has been studied in more than 10,000 patients across 50 clinical trials in various chronic diseases driven by type 2 inflammation.

In addition to the currently approved indications, Sanofi and Regeneron are also studying dupilumab in a broad range of clinical development programs for diseases driven by allergic and other type 2 inflammation, including pediatric asthma (6 to 11 years of age, Phase 3), pediatric atopic dermatitis (6 months to 5 years of age, Phase 2/3), eosinophilic esophagitis (Phase 3), chronic obstructive pulmonary disease (Phase 3), bullous pemphigoid (Phase 3), prurigo nodularis (Phase 3), chronic spontaneous urticaria (Phase 3), and food and environmental allergies (Phase 2).

These potential uses are investigational, and the safety and efficacy have not been evaluated by any regulatory authority. Dupilumab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement.

Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to seven FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, infectious diseases and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically-humanized mice to produce optimized fully-human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
http://www.globenewswire.com/news-release/2020/06/19/2050647/0/en/Dupixent-dupilumab-approved-in-China-for-adults-with-moderate-to-severe-atopic-dermatitis.html?print=1

Novartis discontinues hydroxychloroquine clinical trial

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Novartis discontinues hydroxychloroquine clinical trial

“Novartis discontinues hydroxychloroquine clinical trial based on slow enrollment, remains committed to pandemic research efforts

June 19, 2020: Novartis has made the decision to stop and discontinue its sponsored HCQ clinical trial for COVID-19 due to acute enrollment challenges that have made trial completion infeasible. The recruitment challenge facing our HCQ trial has made it unlikely that the clinical team will be able to collect meaningful data in a reasonable timeframe to determine the effectiveness of HCQ in treating patients with COVID-19. No safety issues have been reported, and there are no conclusions on efficacy from the study.

Novartis will continue to provide supply of HCQ for ongoing investigator-initiated trials (IITs) and upon government requests, as appropriate, where certain conditions are met and the medicine is used in accordance with a nationally endorsed treatment protocol. Researchers at Novartis continue to monitor ongoing guidance from health authorities on the further study of HCQ for COVID-19, as well as the decision by the US Food and Drug Administration to stop the emergency use authorization of HCQ for COVID-19 treatment. In addition, ongoing studies involving other Novartis treatments continue, as well as the newly announced support of a potential vaccine. Novartis also continues to collaborate to find answers for COVID-19 through various partnerships with multi-stakeholder external consortia.

Novartis wishes to thank all of the patients, their families, investigators and healthcare professionals who played a vital part in our HCQ clinical trial (CJWT629A12301, NCT04358081) for COVID-19.”

https://www.novartis.com/news/media-releases/novartis-discontinues-hydroxychloroquine-clinical-trial-based-slow-enrollment-remains-committed-pandemic-research-efforts

Coronavirus (COVID-19) Update: Daily Roundup June 19, 2020

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Coronavirus (COVID-19) Update: Daily Roundup June 19, 2020

June 19, 2020: The U.S. Food and Drug Administration announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:

  • The FDA issued an updated FDA COVID-19 Response At-A-Glance Summary that provides a quick look at facts, figures, and highlights of the agency’s response efforts.
  • The FDA continues to take creative and flexible approaches to address access to critical medical products in response to COVID-19.

    In partnership with academic researchers, non-traditional manufacturers, communities of makers, and individuals who are banding together to support and fill local and national needs, the FDA is actively engaged and is developing ways to support these groups seeking to help their communities.

    Our goal is to help expand the availability of relevant products in ways that are consistent with the FDA’s public-health mission.

    For example, the FDA is working in partnership with the National Institutes of Health (NIH), the Veterans Administration (VA), and America Makes to support non-traditional manufacturing approaches (e.g., 3D printing), to address device shortages including personal protective equipment (PPE).

    Through this partnership, 3D-printable designs for COVID response are assessed by the VA, and the NIH posts them on its 3D Print Exchange. The FDA has, among other things, provided information on labeling and testing for face shields and face masks.

    Today, the FDA web update documents how this partnership has contributed to the number of medical devices — including PPE — and parts available to support the COVID-19 response since its launch 10 weeks ago.

    For example, 31 community-submitted designs passed the testing performed by VA clinics and were given clinically reviewed status.

    In addition, this effort has so far matched more than 272,000 3D-printed face shields and more than 230,000 3D-printed face masks with health care providers and others in need.

    The FDA has issued a temporary policy for face masks and respirators during the COVID-19 public-health emergency.
  • On Tuesday, June 23, 12:00‒1:00 pm ET, the FDA, along with the Centers for Disease Control and Prevention’s (CDC) National Institute for Occupational Safety and Health (NIOSH) and the Occupational Safety and Health Administration (OSHA), will host the second webinar in the respirator webinar series on the topic of Importing Respirators for Health Care Personnel Use during COVID-19 Pandemic.
  • The FDA updated its guidance, titled Notifying CDRH of a Permanent Discontinuance or Interruption in Manufacturing of a Device Under Section 506J of the FD&C Act During the COVID-19 Public Health Emergency, to facilitate notification regarding device manufacturing interruptions or discontinuances.

    The FDA updated this guidance to include a list of device types and corresponding product codes that FDA recommends manufacturers consider in determining whether a notification under Section 506J of the FD&C Act is required during the COVID-19 pandemic.

    The CARES Act, which added Section 506J to the FD&C Act, is an important step that advances FDA’s ability to prevent or mitigate potential medical device shortages during a public health emergency.
  • FDA issued a guidance that provides recommendations to pharmaceutical manufacturers on actions to take when an employee who has been directly involved in manufacturing drugs has a confirmed infection of COVID-19, symptoms of COVID-19, or has been exposed to an infected person. FDA’s recommendations are intended to help avoid negative effects on the safety and quality of drugs.

    FDA expects drug manufacturers to evaluate existing manufacturing controls to prevent drug safety or quality issues related to contamination from SARS-CoV-2.

    Drug manufacturers should also review CDC guidance regarding when employees may continue working following exposure or potential exposure to COVID-19 as well as procedures to minimize exposure and transmission in the workplace.

    FDA is not aware of any drugs that have been contaminated with SARS-CoV-2 or of information indicating transmission of COVID-19 is associated with drugs.
  • The FDA issued a warning letter to one company for selling fraudulent COVID-19 products, as part of the agency’s effort to protect consumers. The warned company, Project 1600, Inc., offers cannabidiol (CBD) products for sale in the United States with misleading claims that the products can mitigate, prevent, treat, or cure COVID-19 in people.

    There are currently no FDA-approved products to prevent or treat COVID-19. Consumers concerned about COVID-19 should consult with their health care provider.
  • The FDA issued a Letter to Clinical Laboratory Staff and Health Care Providers recommending that they stop using COVID-19 antibody tests that are listed on the FDA’s “removed” test list.

    The “removed” test list includes tests in which significant clinical performance problems were identified that cannot be or have not been addressed by the commercial manufacturer in a timely manner, tests for which an Emergency Use Authorization request has not been submitted by a commercial manufacturer of a serology test within a reasonable period of time as outlined in the FDA’s guidance, and tests voluntarily withdrawn by the respective commercial manufacturers.
  • Testing updates:
    • To date, the FDA has authorized 144 tests under EUAs; these include 122 molecular tests, 21 antibody tests, and 1 antigen test.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-daily-roundup-june-19-2020

India approved Glenmark’s Favipiravir for the treatment of mild to moderate COVID-19

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India approved Glenmark’s Favipiravir for the treatment of mild to moderate COVID-19

“Glenmark becomes the first pharmaceutical company in India to receive regulatory approval for oral antiviral Favipiravir, for the treatment of mild to moderate COVID-19

June 20, 2020: In a landmark development for COVID-19 patients in India, Glenmark Pharmaceuticals, a research-led, integrated global pharmaceutical company, today announced the launch of antiviral drug Favipiravir (brand name FabiFlu®) for the treatment of mild to moderate COVID-19 patients.

Glenmark has received manufacturing and marketing approval from India’s drug regulator, making FabiFlu® the first oral Favipiravir-approved medication in India for the treatment of COVID-19.

Favipiravir is backed by strong clinical evidence showing encouraging results in patients with mild to moderate COVID-19.

The antiviral offers broad spectrum RNA virus coverage with clinical improvement noted across age groups 20 to >90 years.

Favipiravir can be used in COVID-19 patients with co-morbid conditions such as diabetes and heart disease with mild to moderate COVID 19 symptoms. It offers rapid reduction in viral load within 4 days and provides faster symptomatic and radiological improvement.

Of most importance, Favipiravir has shown clinical improvement of up to 88% in COVID-19 mild to moderate COVID 19 cases.

Glenmark successfully developed the active pharmaceutical ingredient (API) and the formulation for FabiFlu® through its own in-house R&D team.

Glenmark filed the product for clinical trial with India’s drug regulator DCGI and became the first pharmaceutical company in India to receive approval for conducting phase 3 clinical trial on mild to moderate COVID-19 patients. Commenting on the significance of this development, Mr. Glenn Saldanha, Chairman and Managing Director of Glenmark Pharmaceuticals Ltd., said, “This approval comes at a time when cases in India are spiralling like never before, putting a tremendous pressure on our healthcare system.

We hope the availability of an effective treatment such as FabiFlu® will considerably help assuage this pressure, and offer patients in India a much needed and timely therapy option.”

He added, “FabiFlu® has demonstrated an encouraging response in mild to moderate COVID-19 patients during clinical trials. Moreover, it is orally administered, and so it serves as a more convenient treatment option over other intravenously administered medications.

Glenmark will work closely with the government and medical community to make FabiFlu® quickly accessible to patients across the country.”

Favipiravir is approved in Japan since 2014 for the treatment of novel or re-emerging influenza virus infections. It has a unique mechanism of action: it is converted into an active phosphoribosylated form (favipiravir-RTP) in cells and recognized as a substrate by viral RNA polymerase, thereby inhibiting RNA polymerase activity.

Most patients exhibiting mild to moderate symptoms can benefit from FabiFlu® use. The drug will be available as a prescription-based medication for INR 103/tablet, with recommended dose being 1800 mg twice daily on day 1, followed by 800 mg twice daily up to day 14.

Earlier last month, Glenmark also announced that it is conducting another clinical trial to evaluate the efficacy of two antivirals Favipiravir and Umifenovir as a combination therapy in moderate hospitalized adult COVID-19 patients in India.”
https://www.glenmarkpharma.com/sites/default/files/Glenmark-becomes-the-first-pharmaceut-cal-company-in-India-to-receive.pdf

Roche’s IPATential150 study met one of its co-primary endpoints

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Roche’s IPATential150 study met one of its co-primary endpoints

June 19, 2020: “Roche announced that the phase III IPATential150 study met its co-primary endpoint of radiographic progression-free survival (rPFS) in patients with metastatic castration-resistant prostate cancer (mCRPC) and whose tumours had PTEN loss.

In this patient group, ipatasertib in combination with abiraterone and prednisone/prednisolone provided a statistically significant reduction in the risk of disease worsening or death, compared to current standard of care (abiraterone and prednisone/prednisolone) plus placebo.

The other co-primary endpoint of rPFS in the overall study population (ITT) was not met.

The safety profile for the combination of ipatasertib and abiraterone was consistent with previous analyses and known risks. The results of the IPATential150 study will be presented at an upcoming medical meeting.

While initial data are encouraging, overall survival benefit and additional secondary endpoints are not yet mature.

The trial will continue until the next planned analysis and data will be shared with health authorities.

“Prostate cancer remains a leading cause of death in men worldwide and patients with metastatic castration-resistant prostate cancer can be difficult to treat,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development.

“The early results of the IPATential150 study are encouraging in our ongoing mission to develop new treatment options for people with advanced prostate cancer.”

Ipatasertib is an oral, highly specific, investigational medicine designed to target and bind to all three isoforms of AKT (protein kinase B), which blocks the PI3K/AKT signalling pathway – a key driver of cancer cell growth and proliferation in prostate cancer.

The PI3K/AKT pathway has also been implicated in resistance to anti-androgen therapy as androgen receptor (AR) inhibition is associated with an increase in AKT pathway activation.

Functional loss of the tumour suppressor protein PTEN within the tumour, seen in approximately 40-60% of mCRPC patients, results in hyperactivation of the PI3K/AKT pathway and is associated with adverse outcomes such as increased tumour grade and stage, earlier biochemical recurrence after radical prostatectomy, metastasis, prostate-cancer-specific death, and androgen-independent progression.

Roche’s clinical development programme for ipatasertib focuses on tumours that are frequently found to have activation of the PI3K/AKT pathway.

In addition to prostate cancer, ipatasertib is being studied in certain types of breast cancer including triple-negative breast cancer (TNBC) and hormone-receptor positive (HR+), HER2- negative breast cancer. Results are anticipated later in 2020.

IPATential150 study
IPATential150 is a double-blind, placebo-controlled, randomised phase III study assessing ipatasertib in combination with abiraterone and prednisone/prednisolone, compared to placebo plus abiraterone and prednisone/prednisolone, in adult male patients with asymptomatic or mildly symptomatic, previously untreated mCRPC.

The co-primary endpoints of the study are investigator-determined rPFS in the overall study population, as well as a subpopulation whose tumours have PTEN loss, as assessed by immunohistochemistry (Ventana assay).

PFS in the study is defined as the time from date of randomisation to the first occurrence of disease progression or death from any cause, whichever occurs earlier.

Secondary endpoints include overall survival, safety, time to pain progression, time to initiation of cytotoxic chemotherapy and time to function deterioration.

Ipatasertib
Ipatasertib is an oral, highly specific, investigational medicine designed to target and bind to all three isoforms of AKT, which blocks the PI3K/AKT signalling pathway and may prevent cancer cell growth and survival.

Ipatasertib is being studied in tumours that are frequently found to have activation of the PI3K/AKT pathway, including breast and prostate cancers.

Clinical studies are ongoing to evaluate the efficacy and safety of ipatasertib and the opportunity it may provide to address significant unmet needs for patients with these diseases.

Ipatasertib was discovered at Genentech in partnership with Array BioPharma Inc. (acquired by Pfizer Inc. on July 30, 2019).

Metastatic castration-resistant prostate cancer (mCRPC)
Prostate cancer is the second most frequent cancer and the fifth leading cause of death in men.

Metastatic prostate cancer refers to prostate cancer that has spread beyond the prostate to other parts of the body (metastasised).

Although most men are cured with treatment of localised disease, recurrent or newly diagnosed metastatic disease is associated with significant morbidity and mortality.

Primary treatment of advanced prostate cancer is androgen deprivation therapy (ADT); however, up to one-third of patients will progress despite reduction in serum testosterone levels to castrate levels (<50 ng/dL), either through surgical or medical castration.

Castration-resistant prostate cancer (CRPC) is defined by disease progression, as measured by prostate specific antigen (PSA) or radiographic measures, despite adequate suppression of serum testosterone levels.

Despite the current availability of life-extending therapies for mCRPC, the majority of men will die of their disease: the median life expectancy in this population is less than three years.

Prostate cancer growth and survival is driven by abnormal AR signalling. In CRPC, cell growth and proliferation is also commonly driven by activation of the PI3K/AKT signalling pathway.

In particular, functional loss of the tumour suppressor protein PTEN within the tumour, seen in approximately 40-60% of people with mCRPC, results in hyperactivation of the PI3K/AKT pathway.

PTEN loss is associated with adverse outcomes such as increased tumour grade and stage, earlier biochemical recurrence after radical prostatectomy, metastasis, prostate-cancer-specific death, and androgen-independent progression.

The PI3K/AKT pathway has also been implicated in resistance to anti-androgen therapy as AR inhibition is associated with an increase in AKT pathway activation, suggesting that the tumour compensates for the loss of one pathway with another“
https://www.roche.com/media/releases/med-cor-2020-06-19.htm

FDA Approves First Therapy for Rare Disease

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FDA Approves First Therapy for Rare Disease

June 18, 2020: “The U.S. Food and Drug Administration approved Crysvita (burosumab-twza) injection to treat patients age two and older with tumor-induced osteomalacia (TIO), a rare disease that is characterized by the development of tumors that cause weakened and softened bones.

The tumors associated with TIO release a peptide hormone-like substance known as fibroblast growth factor 23 (FGF23) that lowers phosphate levels.

“Treatment for TIO focuses on identifying and removing the tumor that causes the disease. However, when that is not possible, Crysvita can help increase the levels of phosphate in the blood,” said Theresa E. Kehoe, M.D., acting director of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research.

“As the first FDA-approved therapy to treat this debilitating disease, today’s action is an important step in finding treatment options for patients living with TIO whose tumor cannot be found or removed.”

FGF23 regulates levels of phosphate, an electrolyte that plays important roles in bone maintenance, energy production by cells and nerve function. When there is not enough phosphate in the body, bones begin to soften and weaken, causing osteomalacia (marked softening of bones).

The safety and efficacy of Crysvita were evaluated in two studies that together enrolled 27 adults with TIO.

In both studies, patients received Crysvita every four weeks. For the first study, half of patients achieved normal phosphate levels through week 24 and maintained normal or near normal phosphate levels through week 144.

In the second study, 69% of participants achieved normal phosphate levels through week 24 and maintained normal or near normal phosphate levels through week 88. The results of bone scans for patients in the first study also suggested healing of the bone lesions related to osteomalacia (softening of the bones).

Related News: Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) for the treatment of Tumor-Induced Osteomalacia (TIO)

Hypersensitivity reactions such as rash and hives have been reported in patients who took Crysvita. If serious hypersensitivity reactions occur, patients should stop taking Crysvita and talk with their health care provider about further medical treatment.

Additionally, higher than normal levels of phosphorus may be associated with an increased risk of nephrocalcinosis (a disorder that occurs when too much calcium is deposited in the kidneys).

The most common side effects reported in adults with TIO taking Crysvita were tooth abscess (infection), muscle spasms, dizziness, constipation, injection site reaction, rash and headaches.

Patients taking oral phosphate or active vitamin D, those who have serum phosphate levels within or above the normal range for their age, and patients with severe kidney impairment or end stage renal disease should not take Crysvita.

Crysvita is also FDA-approved to treat adults and children six months and older with X-linked hypophosphatemia, which causes low levels of phosphate in the blood and leads to impaired bone growth and development in children and teenagers.

The FDA granted approval of Crysvita to Ultragenyx Pharmaceutical Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-disease-causes-low-phosphate-blood-levels-bone-softening

Coronavirus (COVID-19) Update: Daily Roundup June 18, 2020

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June 18, 2020: “The U.S. Food and Drug Administration announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:

  • The FDA participated in the International Society for Neglected Tropical Diseases virtual meeting External Link Disclaimer today to discuss how the CURE ID app can be used to capture case reports regarding treatments and advance research into neglected tropical disease and COVID-19.
  • Visit the FDA’s new web section Coronavirus Disease 2019 (COVID-19) Emergency Use Authorizations for Medical Devices.

    This resource provides information on the Emergency Use Authorizations for COVID-19 tests and other medical devices that the FDA has issued related to COVID-19 on individual web pages to help make it easier for the public to access this information.
  • The FDA published FAQs related to its Temporary Policy Regarding Certain Food Labeling Requirements During the COVID-19 Public Health Emergency: Minor Formulation Changes and Vending Machines.
  • Testing updates:
    • To date, FDA has authorized 141 tests under EUAs; these include 120 molecular tests, 20 antibody tests, and 1 antigen test.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-daily-roundup-june-18-2020

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