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USFDA Grants Rare Pediatric Disease Designation to Stealth BioTherapeutics for Elamipretide for the Treatment of Barth Syndrome

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USFDA Grants Rare Pediatric Disease Designation to Stealth BioTherapeutics for Elamipretide for the Treatment of Barth Syndrome

March 3, 2020: Stealth BioTherapeutics announced that the U.S. FDA has granted Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition. Under the RPD classification system of the FDA, the FDA may issue a priority review voucher to a sponsor who receives approval for a “rare pediatric disease,” a severe and life-threatening disease that mainly affects persons aged 18 years and under 200,000 in the United States.

Stealth BioTherapeutics is eligible for a voucher following FDA approval of elamipretide for Barth syndrome, which can be used to receive priority consideration for a subsequent human drug application in complying with the applicable statutory requirements associated with the RPD programme.

Barth syndrome is an ultra-rare genetic condition characterized by cardiac defects that often result in heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed development. Barth syndrome occurs nearly exclusively in males and is estimated to affect one in 200,000 to 400,000 people around the world. There are no FDA- or EMA-approved treatments for Barth syndrome patients at this time.
https://www.streetinsider.com/PRNewswire/FDA+Grants+Rare+Pediatric+Disease+Designation+to+Stealth+BioTherapeutics+for+Elamipretide+for+the+Treatment+of+Barth+Syndrome/16561448.html

FDA Informs Patients, Providers and Manufacturers About Potential Cybersecurity Vulnerabilities in the Certain Medical Devices with Bluetooth Low Energy

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FDA Informs Patients, Providers and Manufacturers About Potential Cybersecurity Vulnerabilities in the Certain Medical Devices with Bluetooth Low Energy

March 03, 2020: U.S. FDA is informing patients, health care providers and manufacturers about a set of cybersecurity vulnerabilities, referred to as “SweynTooth,” that – if exploited – may introduce risks for certain medical devices. SweynTooth affects the wireless communication technology known as Bluetooth Low Energy (BLE).

BLE allows two devices to “connect” and share information in order to perform their intended functions while maintaining the battery life and can be used in medical devices as well as other devices, such as consumer wearables and apps on the Internet of Things. These cybersecurity vulnerabilities may permit an unauthorized user to wirelessly crash the device, stop it from working, or access device functions normally only available to the authorized user.

Up till now, the FDA is not aware of any confirmed adverse events related to these vulnerabilities. However, software to exploit these vulnerabilities in certain situations is publicly available.  FDA is providing additional information regarding the source of these vulnerabilities and recommendations for reducing or avoiding risks the vulnerabilities may pose to a variety of medical devices, such as pacemakers, glucose monitors, and ultrasound devices.

FDA is currently aware of several microchip manufacturers that are affected by these vulnerabilities: Texas Instruments, NXP, Cypress, Dialog Semiconductors, Microchip, STMicroelectronics and Telink Semiconductor. Their microchips may be in a variety of the medical devices, such as those that are implanted in or worn by the patient (such as pacemakers, stimulators, blood glucose monitors and insulin pumps) or larger devices that are in health care facilities (such as electrocardiograms, monitors and diagnostic devices like ultrasound devices).

Medical device manufacturers are already assessing which devices may be affected by the SweynTooth and are identifying risk and remediation actions. Additionally, several microchip manufacturers have already released patches. For more information about SweynTooth cybersecurity vulnerabilities – including a list of the affected devices, see ICS-ALERT-20-063-01 SweynTooth Vulnerabilities, Department of Homeland Security Cybersecurity Infrastructure Security Advisory.

The agency is asking medical device manufacturers in order to communicate to health care providers and patients which medical devices could be affected by SweynTooth and ways to reduce the associated risk. Patients should talk to their health care providers in order to determine if their medical device could be affected and to seek help right away if they think their medical device is not working as expected.

The FDA will continue to evaluate new information about the risk of SweynTooth and will keep the public updated if important new information becomes available.

Furthermore, the FDA will continue its ongoing work with manufacturers and health care delivery organizations—as well as security researchers and other government agencies—to help develop and implement solutions to address cybersecurity issues throughout the device’s total product lifecycle.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, efficiency, 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 protection of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
https://fda.einnews.com/pr_news/511110111/fda-informs-patients-providers-and-manufacturers-about-potential-cybersecurity-vulnerabilities-in-certain-medical-devices-with-bluetooth-low-energy

KemPharm Submits KP415 NDA to USFDA for the Treatment of attention deficit hyperactivity disorder (ADHD)

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KemPharm Submits KP415 NDA to USFDA for the Treatment of attention deficit hyperactivity disorder (ADHD)

March 02, 2020: KemPharm announced that it has submitted a New Drug Application (NDA) under Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act for its investigational product candidate, KP415, to the U.S.FDA. 

KP415 is KemPharm’s product candidate for the treatment of attention deficit hyperactivity disorder (ADHD) which contains serdexmethylphenidate (SDX), KemPharm’s prodrug of the d-methylphenidate (d-MPH). 

At first, the FDA will review the data package and, if deemed to be complete, will issue formal notice of the acceptance of the submission, a process which usually takes sixty (60) days from the date of submission. Since KP415 contains SDX, which is a new molecular entity (NME), the FDA review guidance for the NMEs is ten (10) months from the date of acceptance, which could lead to a potential action (PDUFA) date in March 2021.

“Submission of the KP415 NDA is a significant milestone for KemPharm as we seek FDA approval for our first ADHD product candidate based on our proprietary LAT™ prodrug technology,” said Travis C. Mickle, Ph.D., President and CEO of KemPharm.  “We believe the data package submitted with the NDA supports our conclusion that KP415 is effective in treating ADHD, has an onset of action at 30 minutes, has a  duration of effect of 13 hours, and avoids unnecessary spikes in d-MPH concentrations that may be associated with adverse events.  We also believe that the SDX component of KP415 may have lower abuse potential than relevant d-MPH comparators.” 

The KP415 NDA filing was prepared by KemPharm in collaboration with the Gurnet Point Capital (GPC). KemPharm entered into a strategic licensing agreement in Sep 2019 with an affiliate of GPC, for the exclusive worldwide rights to develop, manufacture and, if approved, commercialize KemPharm’s product candidates containing SDX, including KemPharm’s ADHD product candidates, KP415 and KP484. This license agreement provides that a regulatory milestone payment will be payable to KemPharm thirty (30) days following FDA acceptance of the KP415 NDA. 

“We look forward to working with the FDA as they complete their review of the KP415 NDA,” Dr. Mickle concluded.  “In addition, our work continues with GPC’s commercial team as we now focus on preparing for the potential launch of KP415 in the U.S.”

About KP415: KP415 consists of SDX co-formulated with immediate-release d-MPH and is designed to address unmet needs with the most widely-prescribed methylphenidate ADHD treatments that includes earlier onset of the action and longer duration of therapy, while avoiding unnecessary spikes in d-MPH concentrations that may be associated with adverse events.  Additionally, results from the various Human Abuse Potential trials for the SDX component of KP415 suggest that the prodrug alone may have lower abuse potential than relevant d-MPH comparators.
https://fda.einnews.com/pr_news/511007171/kempharm-submits-kp415-nda-to-the-fda-for-the-treatment-of-adhd

FDA grants Breakthrough Therapy Designation for Roche’s Esbriet (pirfenidone) in unclassifiable interstitial lung disease

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FDA grants Breakthrough Therapy Designation for Roche’s Esbriet (pirfenidone) in unclassifiable interstitial lung disease

March 03, 2020: Roche announced that the U.S.FDA has granted Breakthrough Therapy Designation (BTD) to Esbriet® (pirfenidone) for the adults with unclassifiable interstitial lung disease (uILD).

The designation was granted based on the data from Phase II trial, which studied the efficiency and safety of Esbriet in uILD. The study represented the first randomised controlled trial to entirely enroll patients with progressive fibrosing uILD.

“Today’s milestone for Esbriet builds on our continued commitment to improving the standard of care for people living with fibrotic lung diseases,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development.

“We look forward to discussing the data with the FDA with the hope of bringing our important medicine to those with uILD who are currently without a treatment option.”

“ILD is a term describing primarily a diverse group of more than 200 forms of rare pulmonary diseases. Although ILDs share similar symptoms including cough and shortness of breath, each ILD has different causes, approaches to treatment, and outlooks.

ILD is a term that broadly describes a diverse group of more than 200 types of rare pulmonary diseases. While ILDs share similar features, including cough and shortness of breath, each ILD has different causes, treatment approaches, and outlooks.

Approximately 10 percent of people living with ILD who are examined by a multidisciplinary team can not be offered a definitive diagnosis, even after a thorough investigation, and in such cases patients are classified as having uILD.”


“The Phase II data supporting Breakthrough Designation were just presented as a late-breaking abstract at the 2019 European Respiratory Society’s annual meeting and simultaneously published in The Lancet Respiratory Medicine. The data suggested Esbriet slowed disease progression and supported its efficiency on a number of lung function parameters including forced vital capacity (FVC), in people with uILD. The security and tolerability profile of Esbriet in people with uILD was comparable with that observed in Phase III trials in people with idiopathic pulmonary fibrosis (IPF).

Breakthrough Therapy Designation is designed in order to accelerate the development and review of medicines intended to treat serious or life-threatening conditions with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies. This is the 33rd Breakthrough Therapy Designation for Roche’s portfolio of the medicines.”

“Esbriet is an oral medicine approved for the treatment of IPF and is available in more than 60 countries worldwide. Esbriet has Orphan Drug designation and was approved for use in Europe in 2011 in adults with mild-to-moderate IPF and in the US in people with IPF in October 2014”. https://www.roche.com/investors/updates/inv-update-2020-03-03.htm

Coronavirus (COVID-19) Update: FDA and CDC take action to increase access to the respirators, including N95s, for the health care personnel

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Coronavirus (COVID-19) Update: FDA and CDC take action to increase access to the respirators, including N95s, for the health care personnel

March 3, 2020: The U.S. FDA and the Centers for Disease Control and Prevention took action to make more respirators, including certain N95s, available to health care personnel. Presently, the majority of respirators on the market are indicated for use in industrial settings.

This action allows certain National Institute for Occupational Safety and Health (NIOSH) approved respirators not currently regulated by the FDA to be used in a health care setting by the health care personnel during the coronavirus (COVID-19) outbreak, thereby increasing the number of respirators available to meet the needs of the U.S. health care system.

Respiratory protective devices are designed in order to achieve a very close facial fit and competent filtration of airborne particles. When properly fitted, respirators, such as N95s, can filter more airborne particles than face masks, which is significant during an outbreak of a respiratory disease like COVID-19. The FDA regulates respirators intended for use in a health care setting, on the other hand, most respirators are used in construction and other industrial jobs only and are as a result not required to meet the FDA requirements for testing.

The FDA granted the CDC’s request for an emergency use authorization (EUA) to allow health care personnel in order to use certain industrial respirators during the COVID-19 outbreak in health care settings. The FDA concluded that respirators approved by the NIOSH, but not currently meeting the FDA’s requirements, may be efficient in preventing health care personnel from airborne exposure, including COVID-19, which can cause serious or life-threatening disease that includes severe respiratory illness.

The agencies are not currently aware of extensive shortages of personal protective equipment, but there are reports of the increased ordering of these products and shortages have been observed in some of the U.S. health care institutions. As the COVID-19 epidemic continues to expand worldwide, the FDA and CDC are conscious that the supply chain for these devices will continue to be greatly strained while demand exceeds available supplies. Regional shortages are expected in the circumstances of this emergency. The FDA and CDC are taking steps to address identified and expected shortages by increasing the use of NIOSH-approved respirators but still failing to meet FDA regulatory requirements.

Additionally, while the EUA can help by increase the availability of certain NIOSH-approved respirators to health care personnel, this EUA does not apply to the public, who should not wear these respirators to protect against COVID-19. There is no added health benefit to the general American public to wear a respiratory protective device (such as an N95 respirator). The immediate health risk from COVID-19 is considered low. The CDC recommends everyday preventive actions, such as hand washing, just to help prevent the spread of respiratory diseases.

The FDA will continue to update the public on our efforts to address this outbreak and on identifying solutions to help alleviate any supply chain problems that may occur, including continuing to evaluate the potential impacts of this outbreak on medical device availability and personal protective equipment. We are committed to working with stakeholders across the supply chain to allow access to critical medical products in close collaboration with public health and government partners.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, efficiency, and security of the human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and protection of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
https://fda.einnews.com/pr_news/511023068/coronavirus-covid-19-update-fda-and-cdc-take-action-to-increase-access-to-respirators-including-n95s-for-health-care-personnel

FDA allows labs margin in testing for coronavirus

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FDA allows labs margin in testing for coronavirus

March 1, 2020: The FDA took new steps to expand testing for the coronavirus by allowing certain hospital laboratories in order to use their own tests before being cleared by the agency.

The action followed the complaints from academic medical centres that the previous policy — which required previous approval of the lab tests — was too heavy, too slow and also holding back efforts to diagnose patients.

Experts have warned that the small number of U.S. cases so far may be a reflection of the limited testing, not of the virus’s spread. Testing has been delayed because of problems, now being corrected, involving the only test that had been cleared by the FDA, one created by the Centers for the  Disease Control and Prevention.

Certified hospital laboratories will usually establish their own in-house tests, but the rules of a public health emergency — which now regulate coronavirus outbreak — ensure that these tests need the FDA’s “authorization for emergency use.” Under the policy announced Saturday, the laboratories will start using their own tests after validating them and before the FDA has completed their evaluations.

Therefore, hundreds of labs could soon begin testing thousands of patients.

http://fda.einnews.com/article/510939576?lcf=8DWPqPuUsDVNDakfEIxsCA%3D%3D

USFDA Approves First Generic of Daraprim

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USFDA Approves First Generic of Daraprim

Feb 28, 2020: The U.S. FDA  has approved an application for the first generic of Daraprim (pyrimethamine) tablets for the treatment of toxoplasmosis (an infection caused by the parasite Toxoplasma gondii) when used with a sulfonamide (a group of medicines used to treat bacterial infections).

Toxoplasmosis is an infection caused by a single-celled parasite named Toxoplasma gondii that can cause brain, eye or other organ damage if severe. A toxoplasma infection can occur, inter alia, by eating undercooked, contaminated meat or shellfish; drinking water contaminated with toxoplasma; or by accidental swallowing of the parasite through contact with toxoplasma-containing cat feces. It is considered the leading cause of death in the United States due to foodborne disease.

In pregnant women and people with weak immune systems, such as those with HIV or AIDS, those undergoing other forms of chemotherapy and those who have recently received an organ transplant, serious toxoplasmosis is more likely. However, even people with healthy immune systems can occasionally experience toxoplasmosis damage to the eyes.One focus area under the action plan is to improve the efficiency of the generic drug production, review and approval process, and to close loopholes that allow brand-name drug companies to avoid generic competition.

 As part of these important efforts, the FDA maintains a list of off-patent, off-exclusivity drug products without an approved generic in order to improve transparency and support the development and submission of applications for drugs with limited competition. Pyrimethamine is incorporated on this list. The FDA also prioritizes the review of the submissions for generic drugs for which there are fewer than three approved generic versions for the reference listed drug (RLD) and for which there are no blocking patents or exclusivities on the RLD.

In addition, certain “gaming” tactics have been used at times to delay generic competition. One example is when brand-name drug manufacturers attempt to prevent potential generic applicants from obtaining samples of the certain medicines necessary to support approval of a generic drug application. To increase clarity on this subject, the FDA posted a list describing all drugs for which a prospective generic applicant has received an inquiry concerning the limited distribution of the reference drug. Daraprim appears on this chart.

The most common side effects for pyrimethamine include hypersensitivity reactions (immune responses) that can irregularly be severe, such as Stevens-Johnson syndrome and toxic epidermal necrolysis (two forms of the same life-threatening disease that causes rash, skin peeling and sores on the mucous membranes), erythema multiforme (a skin disorder with bulls-eye-shaped lesions), anaphylaxis (a severe allergic reaction that may include difficulty breathing and shock) and hyperphenylalaninemia (elevated concentration of the amino acid phenylalanine in the blood), particularly when pyrimethamine is administered at the same time as a sulfonamide.

“Pyrimethamine should not be used in patients with known hypersensitivity to pyrimethamine or with documented megaloblastic anemia because of folate (a naturally-occurring B vitamin) deficiency. Women who takes pyrimethamine should not become pregnant. Patients should keep pyrimethamine out of the reach of children.”

A small “starting” dose for toxoplasmosis is recommended in patients with convulsive disorders in order to avoid the potential nervous system toxicity of pyrimethamine. Pyrimethamine should be used with caution in patients with impaired renal (kidney) or hepatic (liver) function or in patients with possible folate deficiency, such as individuals with malabsorption syndrome, alcoholism or pregnancy, and those receiving therapy, such as phenytoin (an anti-epileptic drug), affecting folate levels.

The sponsor of the approved generic version of the Daraprim Tablets is Cerovene Inc.

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

USFDA accepts Roche’s Biologics License Application for fixed-dose subcutaneous combination of Perjeta and Herceptin for the treatment of HER2-positive breast cancer

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USFDA accepts Roche’s Biologics License Application for fixed-dose subcutaneous combination of Perjeta and Herceptin for the treatment of HER2-positive breast cancer

Feb 25, 2020: USFDA has accepts Roche’s Biologics License Application (BLA) for the fixed-dose combination (FDC) of Perjeta® (pertuzumab) and Herceptin® (trastuzumab) with hyaluronidase, administered by subcutaneous injection in combination with intravenous (IV) chemotherapy, for the treatment of eligible patients with HER2-positive breast cancer.

The BLA for the FDC is based on results from the Phase III study FeDeriCa, which demonstrated non-inferior levels of Perjeta in the blood (pharmacokinetics) and comparable efficacy and safety with Perjeta plus Herceptin and chemotherapy standard IV infusions. The FDC SC administration takes about eight minutes for the initial loading dose, and about five minutes for each subsequent maintenance dose.This is opposed to about 150 minutes for infusion using the normal IV formulations of a loading dose of Perjeta and Herceptin, and about 60-150 minutes for subsequent maintenance infusions of the two medicines.

The study of FeDeriCa reached its primary endpoint, with FDC SC administration showing non-inferior levels of Perjeta in the blood at a given dose interval (Ctrough) as opposed to Perjeta IV. A secondary endpoint of non-inferior Ctrough of Herceptin was also met, for the study to ensure that people were receiving sufficient dosing with Perjeta and Herceptin as compared to the established IV doses at the same treatment intervals. Additionally, rates of total pathological complete response (pCR), another secondary endpoint, were comparable between the treatment arms. The FDC’s safety profile in conjunction with chemotherapy was comparable with that of Perjeta’s IV administration plus Herceptin and chemotherapy and no new safety signals were found, including no significant difference in heart toxicity. Alopecia, diarrhoea, diarrhoea, and anaemia were the most common adverse events in both armrests.
In previous studies of other SC formulations, it has been shown that SC administration is strongly preferred by the majority of patients compared with IV administration of the same drug, with the most common reason being that administration in the clinic needed less time.

In the PHranceSCa report, Roche is currently investigating patient preference for FDC SC administration in humans with HER2-positive early breast cancer (eBC) compared to standard IV administration of Perjeta and Herceptin. Interim findings of this Phase II study will be discussed at a medical conference to come.
https://www.roche.com/media/releases/med-cor-2020-02-25.htm

Sanofi to create new industry-leading European company to provide active pharmaceutical ingredients

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Sanofi to create new industry-leading European company to provide active pharmaceutical ingredients

Feb 24, 2020: Sanofi plans to produce a major leading European company dedicated to the production and marketing to the third parties of active pharmaceutical ingredients (API), which are the important molecules responsible for the valuable effects used in the composition of any drug.

The project consists of creating a separate company which would combine Sanofi’s API commercial and development activities with six of its European API production sites : Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France).

With growing medicine shortages that significantly impact patient care, the new thing would contribute to supporting and securing API manufacturing as well as supply capacities for Europe and beyond. In Europe, the new API industry champion is expected in order to help in balancing the industry’s heavy reliance on API sourced from the Asian region.

With approximately € 1 billion in expected sales by 2022, the new company will rank as the world’s second-largest API firm. It is planned to hire 3,100 skilled staff and be headquartered in France. A proposed IPO on Euronext Paris would be evaluated according to market conditions, with a decision anticipated by 2022.

The new entity plans to increase its sales to third parties by operating independently and to extend its collaborations with other pharmaceutical companies in order to take advantage of new growth opportunities and have the ability to adapt deftly to customer needs. The new company would be exclusively positioned to benefit from its important competitive strengths, including a broad portfolio of both volume and niche products, high standards of quality, competitive pricing, state-of-the-art industrial capabilities and technologies across Europe (including France, Italy, Germany, Hungary, and the United Kingdom) as well as purchase an extensive commercial network covering more than 80 countries. 

Sanofi is fully dedicated to the new company’s success and intends to establish a long-term customer relationship with the new API supplier and to hold an alternative stake of about 30% in the new company. To provide the optimal conditions for success, Sanofi intends the new company to be debt-free in order to maximize its future investment capacities, and is committed to outstanding an important customer.
https://www.sanofi.com/en/media-room/press-releases/2020/2020-02-24-16-03-59

AstraZeneca divests global rights to Movantik (naloxegol)

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AstraZeneca divests global rights to Movantik (naloxegol)

Feb 25, 2020: AstraZeneca has decided to sublicense its global rights to Movantik (naloxegol), excluding Europe, Canada and Israel, to RedHill Biopharma (RedHill). Movantik is a peripherally acting mu-opioid receptor antagonist (PAMORA) and are indicated for the treatment of opioid-induced constipation (OIC).

Ruud Dobber, Executive Vice President, BioPharmaceuticals Business Unit, said: “This divestment supports our strategy of realizing value from our portfolio of medicines that are mature or outside of our current scope to allow reinvestment in our main therapy areas. Movantik is an important established medicine and RedHill’s divestment will ensure its continued patient availability.”

According to the agreement, AstraZeneca will continue to manufacture and supply Movantik to RedHill during a transition period. In 2015, AstraZeneca entered into a co-commercialisation conformity with Daiichi Sankyo, Inc. for Movantik in the US, which will be transferred to RedHill.

Financial considerations: RedHill will make an direct payment of $52.5m to AstraZeneca on closing and a further non-contingent payment of $15m in 2021. Incomes arising from the upfront payment, balance by a charge for derecognition of the associated intangible asset, and the future payment will be reported in AstraZeneca’s financial statements in Other Operating Income & Expense. In 2019, Movantik generated sales of $96m in the US. The divestment is expected to complete in the first quarter of 2020, subject to expected closing conditions and regulatory clearances. After completion, the agreement will not impact the Company’s financial guidance for 2020.

Movantik: Movantik is a once-daily oral PAMORA approved by the US FDA for the treatment of OIC in the adult patients with chronic non-cancer pain. Movantik was licensed from the Nektar Therapeutics in 2009. In 2016, AstraZeneca divested the rights in Europe to ProStrakan Group (now KKI) and the rights in Canada and Israel to the Knight Therapeutics.
https://www.astrazeneca.com/media-centre/press-releases/2020/astrazeneca-divests-global-rights-to-movantik-25022020.html

Y-mAbs Announces Positive Pre-Biologics License Application Meeting with FDA for Omburtamab

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Y-mAbs Announces Positive Pre-Biologics License Application Meeting with FDA for Omburtamab

Feb. 26, 2020: Therapeutics announced that it has completed a positive Type B Pre-Biologics License Application (“Pre-BLA”) meeting with the U.S.FDA regarding a potential pathway for FDA approval of omburtamab for the treatment of patients with CNS/leptomeningeal metastases from neuroblastoma.

At the pre-BLA meeting, the Company reached placement with the FDA on an Accelerated Approval Pathway for omburtamab along with the rolling BLA submission. The Company expects to complete the rolling BLA in about 10 weeks.

“Under omburtamab’s breakthrough therapy designation, omburtamab qualifies for the rolling BLA submission, which allows for individual modules of the application to be submitted by Company and reviewed by the FDA on a rolling basis, rather than waiting for all sections of the BLA application to be completed before submission.”

“As formerly announced data readout from a single-center study (Study 03-133), at Memorial Sloan Kettering Cancer Center (“MSK”) where the 107 evaluable patients with the CNS/leptomeningeal metastases from neuroblastoma received up to two doses of the radiolabeled omburtamab and showed that the patients had a median survival of 50.8 months, with the final median survival not yet being reached. The Company plans to announce the complete clinical data package later this year. Additionally, the Company is planning for the submission of a Marketing Authorization Application in Europe in the fourth quarter this year.

Researchers at the MSK developed the omburtamab antibody, which is completely licensed by MSK to Y-mAbs. Consequently, MSK has institutional financial interests in the product and in Y-mAbs”.

About Y-mAbs: “Y-mAbs is a late-stage clinical biopharmaceutical company that focus on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer. The Company has a broad and advanced product pipeline that includes two pivotal-stage product candidates—naxitamab and omburtamab—which target tumors that express GD2 and B7-H3, correspondingly.”
https://fda.einnews.com/pr_news/510656396/y-mabs-announces-positive-pre-bla-meeting-with-fda-for-omburtamab

NIH clinical trial of remdesivir to treat coronavirus disease 2019 (COVID-19) begins

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NIH clinical trial of remdesivir to treat coronavirus disease 2019 (COVID-19) begins

Feb 25, 2020: A randomized, controlled clinical trial to evaluate the safety and effectiveness of the investigational antiviral remdesivir in hospitalized adults diagnosed with coronavirus disease 2019 (COVID-19) has begun at University of Nebraska Medical Center (UNMC) in Omaha. The trial regulatory sponsor is the National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH.

This is the first clinical trial in the United States in order to evaluate an experimental treatment for COVID-19, the respiratory disease first detected in December 2019 in Wuhan, Hubei Province, China.

The first participant in the trial is an American who was repatriated after being quarantined on the cruise ship Diamond Princess that docked in Yokohama, Japan and volunteered to participate in the study. The study can be adapted to evaluate additional investigative therapies and enroll participants at other sites in the United States and around the world.

The Food and Drug Administration (FDA) has not approved specific therapies for treating people with COVID-19, the disease caused by the newly emerging SARS-CoV-2 virus (formerly known as 2019-nCoV). Infection may cause mild to severe respiratory disease, and symptoms may include fever, cough, and breathing shortness. The World Health Organization (WHO) reported 77,262 confirmed cases of COVID-19 and 2,595 deaths in China as of 24 February, and 2,069 cases of COVID-19 and 23 deaths in 29 other countries.

According to the Centers for Disease Control and Prevention (CDC), there were 14 confirmed cases of COVID-19 reported in the United States, and an estimated 39 cases among people repatriated to the United States.Remdesivir, developed by Gilead Sciences Inc., is an investigational broad-spectrum antiviral treatment. It was previously tested in humans with Ebola virus disease and has shown promise in animal models for treating Middle East respiratory syndrome (MERS) and severe acute respiratory syndrome (SARS), which are caused by other coronaviruses.

Remdesivir clinical trials are underway in China, too. In line with the consultations convened by the WHO on the development of a therapeutic trial for patients with COVID-19, NIAID developed the current study taking into account those designs. Participants in the NIH-sponsored trial must have laboratory-confirmed SARS-CoV-2 infection and evidence of lung involvement, including rattling sounds when breathing (rales) requiring additional oxygen or abnormal X-rays in the chest, or mechanical ventilation disease. The study will not include individuals with reported infection who have mild, cold-like symptoms or no apparent symptoms.

Eligible patients will provide informed consent to participate in the trial, in accordance with standard clinical research protocols. On the first day of admission to the study, participants in the investigational therapy group will receive 200 milligrams (mg) of remdesivir intravenously. For the duration of the hospitalization, they will receive another 100 mg each day, for a total of up to 10 days. The placebo group will obtain a solution that resembles remdesivir, which contains only inactive ingredients, at an equal volume.
For more information, visit ClinicalTrials.gov and search identifier NCT04280705. https://www.nih.gov/news-events/news-releases/nih-clinical-trial-remdesivir-treat-covid-19-begins

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