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FDA grant approval for GSK’s Voltaren Arthritis Pain for over-the-counter use in the United States

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FDA grant approval for GSK’s Voltaren Arthritis Pain for over-the-counter use in the United States

Feb 17, 2020: GlaxoSmithKline announced that the U.S.FDA has approved Voltaren Arthritis Pain (diclofenac sodium topical gel, 1% (NSAID)- arthritis pain reliever) as an over-the-counter (OTC) product for the temporary relief of arthritis pain in the wrist, elbow, hand, foot, ankle or knee in adults (18 years and older).  

After the FDA’s approval, Voltaren Arthritis Pain becomes the first and the only prescription-strength, nonsteroidal anti-inflammatory (NSAID) topical gel for arthritis pain accessible OTC in the United States.

“Voltaren Gel, to whom GSKCH has the rights, is currently available in the US only with a prescription. Voltaren Arthritis Pain’s OTC approval today will provide over – the counter access to this topical treatment option for the nearly 30 million Americans with osteoarthritis. 

Franck Riot, Head of R&D, GSK Consumer Healthcare said: “For the millions of the people around the world living with arthritis, joint pain and stiffness are everyday realities. At GSK, we are committed to getting better the quality of life of these people and today’s approval is progress towards this, providing consumers in the US with increased right to use an effective, proven arthritis pain relief option.

At present Voltaren is the number 1- OTC topical pain relief brand globally, and we look forward to expanding its accessibility in the US.”

Additionally, the Osteoarthritis Research Society International (OARSI)  just updated and expanded their new guidelines for non-surgical management of OA by developing patient-focused treatment recommendations. These updated guidelines powerfully recommend topical non-steroidal anti-inflammatory drugs (NSAIDs) for persons with knee OA.  Of the non-core interventions measured as part of this guideline update, topical NSAIDs were suggested more strongly than all oral analgesics due to favourable balance of consistent effectiveness and minor transient side effects. The submission in support of the Voltaren Arthritis Pain approval today was based on clinical evidence in hand and knee OA supporting the initial drug approval. The data showed a clear and appreciable progression of pain relief starting as early as week 1 (as demonstrated by significant pain symptom changes across multiple pre-specified endpoints).

About Voltaren Arthritis Pain: The active ingredient in Voltaren Arthritis Pain, diclofenac sodium, is an effective medicine that is clinically proven in order to relieve joint pain due to arthritis. Voltaren Arthritis Pain penetrates through the skin at the application site in order to deliver arthritis pain relief. Voltaren Arthritis Pain offers patient who suffers from OA a different option to oral analgesics.  It targets pain directly at the site and amount of the diclofenac sodium that is systemically absorbed from the Voltaren Arthritis Pain is on average 6% of the systemic exposure from an oral form of the diclofenac sodium. GSK anticipates Voltaren Arthritis Pain will be accessible on US shelves in Spring 2020”. For more information, visit www.VoltarenGel.com.
https://us.gsk.com/en-us/media/press-releases/fda-approves-gsk-s-voltaren-arthritis-pain-for-over-the-counter-use-in-the-united-states/

Health ministry issues notification to regulate all the medical devices under D&C Act from April 1, 2020

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Health ministry issues notification to regulate all the medical devices under D&C Act from April 1, 2020

Feb 11, 2020: Union health ministry has notified that all the medical devices will nowadays regulated under the Drugs and Cosmetics (D&C) Act, 1940 with effect from the April 1, 2020 for efficient compliance.

“The ministry after consultation with the Drugs Technical Advisory Board (DTAB) has further notified to amend the Medical Devices Rules, 2017 and precised that these rules may be called the Medical Devices (Amendment) Rules, 2020 and will come into force on April 1, 2020.


According to a Gazette Notification dated February 11, 2020, after consultation with the DTAB, the health ministry hereby specifies that all the medical devices that are intended for use in human beings or animals will be regulated as drugs with effect from the April 1, 2020, which includes all the devices including an instrument, apparatus, implant, appliance, material or other article, whether used alone or in combination, also including a software or an accessory, intended by its producer to be used particularly for the human beings or animals which does not attain the primary intended action in or on human body or animals by any the pharmacological or immunological or metabolic means, but which may help in its intended function by such means for one or more of the specific purposes of ?(i) diagnosis, prevention, monitoring, treatment or alleviation of any the disease or disorder; (ii) diagnosis, monitoring, treatment, alleviation and assistance for, any injury or disability; (iii) investigation, replacement or modification or support of anatomy or of a physiological process; (iv) supporting or satisfying life; (v) disinfection of the medical devices; and (vi) control of the conception.”
The notification further states that “In the Medical Devices Rules, 2017 (here in after to be referred as said Rules), after Chapter III, the following Chapter IIIA will be inserted, namely-“Chapter IIIA Registration of certain Medical Devices 19a. This Chapter shall be applicable to all devices notified under clause (b) of section 3 of the Act except the medical devices and devices specified in the Eighth Schedule of these rules. The Medical devices referred to in sub-rule (1) shall be registered with the Central Licensing Authority through an identified online portal established by the Central Drugs Standard Control Organisation (CDSCO) for this purpose.

The notification also states that any person who imports any medical device referred in rule 19A shall upload the following information relating to that medical device for registration on the “Online System for Medical Devices” established by the CDSCO for this purpose. The importer shall upload the name of the company or firm or any other entity importing the medical device and specification and standards of that medical device and also details of the medical device. Welcoming the move, Pavan Choudary, chairman and director-general, Medical Technology Association of India (MTaI) said, “We welcome the government’s decision to regulate all devices. These regulations, that are a continuation of the Medical Devices Rules 2017, which were launched after meticulous deliberations for 2 years, are in line with the government’s vision to provide equitable access to quality healthcare. It is also noteworthy that health ministry is recruiting competent resources to cater to the additional workload that these new regulations will bring.”

file:///E:/website%20matter/Gsr102eRegistration%20of%20certain%20medical%20devices.pdf

New Phase IIIb Interim Data from the STARDUST Study Shows about Two-Thirds of Patients with Moderately to Severely Active Crohn’s Disease Achieved Clinical Remission After Two Doses of the STELARA® (ustekinumab)

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New Phase IIIb Interim Data from the STARDUST Study Shows about Two-Thirds of Patients with Moderately to Severely Active Crohn’s Disease Achieved Clinical Remission After Two Doses of the STELARA® (ustekinumab)

Feb. 14, 2020: The Janssen Pharmaceutical Companies of Johnson & Johnson announced temporary data from the Phase IIIb STARDUST study. At week 16, 79 per cent of the patients with moderately to severely active Crohn’s disease (CD) achieved clinical response1 and 67 per cent were in clinical cutback after receiving one ~6 mg/kg IV dose followed by one 90 mg SC dose of the STELARA® (ustekinumab), open-label.

Intestinal ultrasound (IUS) responses were considered and were detected as early as week 4. Week 16 data (digital oral presentation, or DOP 13) and IUS response data (DOP 10) from STARDUST, also presented as digital oral presentations at the 15th Congress of the European Crohn’s & Colitis Organization (ECCO).

The primary endpoint of the 48-week STARDUST study is relative endoscopic response among all adult patients with CD receiving STELARA maintenance therapy.

At week 16, patients who attained ≥70 point decrease in Crohn’s Disease Activity Index score (CDAI70 responders) were randomized into the treat-to-target or routine standard of care treatment groups at the 1:1 ratio.

“Of the 220 CDAI70 responders randomized to treat-to-target arm, 37 percent attained endoscopic response at the week 16.

Endoscopy at week 16 was measured only in the treat-to-target group. Treat-to-target is the practical treatment strategy where regularly monitored outcomes, like endoscopic response, biomarkers and clinical symptoms, guide use of the medication. 

STARDUST is the first study of a treat-to-target strategy in the CD using endoscopic response to guide treatment.

IUS is a corresponding method of assessing CD activity which is based upon measuring transmural bowel features, like the thickness of the bowel wall and presence of the hypervascularization. STARDUST is the first study in order to use IUS for monitoring CD patients in an interventional setting. Prospect studies need to verify whether early IUS response at week 4 is predictive of longer-term (i.e., week 16 and up to week 48) clinical and endoscopic outcomes for the CD patients. Janssen is presenting a total of the 23 abstracts at this year in ECCO congress. At present STELARA is approved for the treatment of adults with moderately to severely active Crohn’s disease in the U.S., Canada, EU and Japan.

About STARDUST Trial
STARDUST is a randomized, international, multicenter, interventional Phase IIIb study assessing the proportion of patients with endoscopic response, identified as a 50% reduction in simple endoscopic score of Crohn’s disease (SES-CD) at week 48 from baseline. STARDUST is evaluating 500 participants receiving an IV induction dose of STELARA 6 mg/kg, followed by a week 8 injection of STELARA 90 mg SC. At week 16, patients with a 70 point CDAI reduction (CDAI70) were randomized to dose-to target or standard arms (1:1 ratio) and followed through the end of the study (48 weeks).

About STELARA® (ustekinumab)
STELARA® (ustekinumab), a human IL-12 and IL-23 antagonist, is accepted in the United States for the treatment of:
 1) Adults and children (12 years) and older with moderate to severe plaque psoriasis and are candidates for the phototherapy or systemic therapy;
2) Adult patients (18 years or older) who have active psoriatic arthritis, used alone or in combination with the methotrexate (MTX);
3) Adult patients (18 years and older) with moderately to harshly active Crohn’s disease;
4) Adult patients (18 years and older) with moderately to harshly active ulcerative colitis.

STELARA Dosing for Crohn’s Disease
Adults with Crohn’s disease will receive the first dose of STELARA through a vein in the arm (intravenous infusion) in a healthcare facility by a healthcare provider. It takes at least 1 hour to receive the full dose of medicine. STELARA will then be given as an injection under the skin (subcutaneous injection) 8 weeks after the first dose of STELARA, then every 8 weeks thereafter. See full Prescribing Information for Dosing Information for other indications. The Janssen Pharmaceutical Companies of Johnson & Johnson maintain exclusive worldwide marketing rights to STELARA®.

IMPORTANT SAFETY INFORMATION
STELARA® is a prescription medicine that can affect your immune system. STELARA® have serious side effects including:

Serious Infections
It lowers the ability to fight infections and may increase the risk of infections. While taking STELARA®, patients may suffer from serious infections, which may require hospitalization, including tuberculosis (TB), and infections caused by bacteria, fungi, or viruses.

Before starting STELARA®, tell your doctor if you:
think you have any type of infection or have symptoms of an infection such as:
fever, sweats, or chills
muscle aches
cough
feel very tired
weight loss
shortness of breath
blood in phlegm
warm, red, or painful skin or sores on your body
diarrhea or stomach pain
burning when you urinate or urinate more often than normal are being treated for an infection.
get a lot of infections or have infections that keep coming back.

 “After starting STELARA®, call your doctor right away if you have any symptoms of an infection (see above). People who have a genetic problem where the body does not make any of the proteins interleukin 12 (IL‐12) and the interleukin 23 (IL‐23) are at a higher risk for the certain serious infections that can spread throughout the body and cause death. People who take STELARA® may also get these infections”.

Cancers
STELARA® can decrease the activity of your immune system and add to your risk for certain types of cancer. Tell your doctor if you ever had any type of cancer. Some people who had risk factors for skin cancer can develop certain types of skin cancers while receiving STELARA®. Tell your doctor if you observe any new skin growths.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS is a rare condition that can affect the brain and can cause death. If RPLS can detect early and treated, most people can recover.

Serious Allergic Reactions: If you have any symptoms of a serious allergic reaction such as: feeling faint, swelling of your face, eyelids, tongue, or throat, chest tightness, or skin rash then stop using STELARA® and get medical help right away

Lung Inflammation: Cases of lung inflammation can occur in some people who receive STELARA® and may be serious. You should not receive the BCG vaccine during one year before receiving the STELARA® or one year after you stop receiving STELARA®. Tell your doctor about all the medicines you take, including prescription and all other medicines, vitamins, and herbal supplements.

When prescribed STELARA®: Use STELARA® exactly as your doctor prescribed you.STELARA® is intended for the use under the guidance and supervision of your doctor. In children 12 years and older, it is suggested that STELARA® can be administered by a healthcare provider. Common side effects of STELARA® include: nasal congestion, and runny nose, sore throat, upper respiratory infections, fever, headache, tiredness, nausea, itching and vomiting, redness at the injection site, urinary tract infections, vaginal yeast infections, sinus infection, stomach pain, diarrhoea, and joint pain. These are not all of the possible side effects with STELARA®. Discuss with your doctor about any side effect that you experience.

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”.
https://www.janssen.com/new-phase-3b-interim-data-stardust-study-show-two-thirds-patients-moderately-severely-active-crohns

Deciphera Pharmaceuticals Announces U.S. FDA Acceptance of NDA and Priority Review for Ripretinib in the Patients with Advanced Gastrointestinal Stromal Tumors

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Deciphera Pharmaceuticals Announces U.S. FDA Acceptance of NDA and Priority Review for Ripretinib in the Patients with Advanced Gastrointestinal Stromal Tumors

Feb. 12, 2020: Deciphera Pharmaceuticals announced that the U.S. FDA has accepted for Priority Review its NDA seeking approval for ripretinib, the Company’s investigational broad-spectrum KIT and PDGFRα inhibitor, for the treatment of patients with advanced gastrointestinal stromal tumors (GIST).

The FDA granted Priority Review for the NDA, which provides for a six-month review, and allocated a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 13, 2020.

“The FDA’s acceptance of our NDA conveys us one step nearer to our goal of providing patients with the advanced GIST a potential new treatment option.”

Priority Review designation is for drugs that, if approved, would be considerable improvements in the safety or efficiency of the treatment, diagnosis, or prevention of the serious conditions when compared to the standard applications.

Under the PDUFA, a Priority Review targets a review time of six months compared to a standard review time of the ten months. in the past, FDA  granted Breakthrough Therapy Designation (BTD) for the ripretinib for treatment of the patients with advanced GIST ,received prior treatment with imatinib, sunitinib, and regorafenib.

BTD is designed in order to expedite the development and review of drugs that are projected to treat a serious condition and preliminary clinical evidence indicates that the drug may reveal substantial improvement above available therapy.

The FDA is evaluating the NDA under the pilot program of the Oncology Center of Excellence for Real Time Oncology Review (RTOR).

This pilot program seeks to create a more reliable evaluation process to ensure that patients have access to safe and effective treatments as soon as possible, while preserving and improving the quality of the review.

You can find further information on RTOR at: https:/www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program.

Additionally, the Company has submitted additional marketing applicationsin Canada and Australia Australia for ripretinib in the advanced GIST. These submission are part of the FDA’s pilot program for the Project Orbis, and both received designations for priority review. The Project Orbis pilot programme, an initiative of the FDA Oncology Center of Excellence, is designed to provide a framework for the simultaneous submission and review among international partners of oncology products.You will find additional information concerning Project Orbis at: https://www.fda.gov/about-fda/oncology-center-excellence/project-orbis.

The NDA submission is supported by constructive results from the Company’s INVICTUS pivotal Phase 3 study of the ripretinib in advanced GIST. INVICTUS is a randomized (2:1), double-blind, placebo-controlled, international, multicenter study designed in order to evaluate the effectiveness and safety of ripretinib compared to placebo in 129 patients with the advanced GIST whose preceding therapies have included at least imatinib, sunitinib, and regorafenib. As per the report, the study completed its primary endpoint of improved progression-free survival compared to placebo in the patients with fourth-line and fourth-line plus GIST, as determined by the blinded independent central radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.

Ripretinib is an investigational tyrosine kinase switch control inhibitor that was designed to broadly inhibit KIT and PDGFRα mutated kinases by using the unique dual mechanism of action that controls the kinase switch pocket and activation loop. Ripretinib granted Fast Track Designation and Breakthrough Therapy Designation by the FDA for the treatment of the patients with advanced GIST, who have received previous treatment with imatinib, sunitinib, and regorafenib. The FDA also granted Priority Review for the NDA for the ripretinib, and allowed a PDUFA target action date of Aug. 13, 2020. Also, ripretinib granted Orphan Drug Designation for the treatment of GIST by the FDA and European Medicines Agency (EMA). For more information concerning the Company’s clinical trials with ripretinib, please visit www.clinicaltrials.gov.
https://investors.deciphera.com/news-releases/news-release-details/deciphera-pharmaceuticals-announces-us-food-and-drug

U.S.FDA Accepts for Priority Review Bristol-Myers Squibb’s Biologics License Application for the Lisocabtagene Maraleucel (liso-cel) for the Adult Patients with Relapsed or Refractory Large B-Cell Lymphoma

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U.S.FDA Accepts for Priority Review Bristol-Myers Squibb’s Biologics License Application for the Lisocabtagene Maraleucel (liso-cel) for the Adult Patients with Relapsed or Refractory Large B-Cell Lymphoma

Feb 13, 2020: U.S.FDA accepted for the Priority Review its Biologics License Application (BLA) for BMS’s lisocabtagene maraleucel (liso-cel), the company’s autologous anti-CD19 chimeric antigen receptor (CAR) T-cell immunotherapy with a defined composition of purified CD8+ and CD4+ CAR T cells for the treatment of adult with relapsed or refractory (R/R) large B-cell lymphoma after at least two prior therapies.

A Prescription Drug User Fee Act (PDUFA) set by FDA as goal date of August 17, 2020.

The BLA, submitted by the Juno Therapeutics, a completely owned subsidiary of BMS, is based on the safety and efficient results from the TRANSCEND NHL 001 trial, evaluating liso-cel in 268 patients with the R/R large B-cell lymphoma, that include diffuse large B-cell lymphoma (DLBCL), high-grade lymphoma, primary mediastinal B-cell lymphoma and Grade 3B follicular lymphoma.

TRANSCEND NHL 001 is the major study of CD19-directed CAR T cells in order to support a BLA to date and was the new subject of an oral presentation at the 61st American Society of Hematology Annual Meeting and Exposition. A priority review classification, according to the FDA, would concentrate overall attention and resources on reviewing drug applications that, if approved, will substantially improve the
safety or efficacy of treatment, diagnosis, or prevention of severe conditions compared to standard applications. Previously, Liso cel was granted FDA Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations for active R / R major B-cell non Hodgkin lymphoma, like DLBCL, not otherwise defined (de novo or transformed from indolent lymphoma), PMBCL or Grade 3B FL, and Priority Medicines (PRIME) by the European Medicines Agency for R / R DLBCL.
https://news.bms.com/press-release/us-food-and-drug-administration-fda-accepts-priority-review-bristol-myers-squibbs-biol

Merck’s Checkpoint Inhibitor Keytruda (pembrolizumab) Hits Another Mark in Breast Cancer

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Merck’s Checkpoint Inhibitor Keytruda (pembrolizumab) Hits Another Mark in Breast Cancer

Feb 12, 2020: Merck’s checkpoint inhibitor Keytruda (pembrolizumab) proved itself efficient in yet another cancer indication. The company announced positive results from the pivotal Phase III KEYNOTE-355 trial of the Keytruda in combination with chemotherapy in patients with metastatic triple-negative breast cancer (mTNBC) whose tumors uttered PD-L1.

The trial met one of its twin primary endpoints, progression-free survival (PFS). An independent Data Monitoring Committee (DMC) performed interim review of the study, showing that first-line treatment with Keytruda with nab paclitaxel, paclitaxel orgemcitabine/ carboplatin had a statistically significant and clinically significant improvement in PFS relative to chemotherapy alone.

The DMC suggested the trial continue without changes in order to evaluate other dual primary endpoints, which is generally survival (OS).
KETNOYE 355 is a two part Phase III trial which is randomized.Part 1 was openlabel and
 lookedat Keytruda’s protection and tolerability in 30 patients with either nabpaclitaxel, paclitaxel, or gemcitabine/carboplatin.Part 2 was doubleblinded, with double primary OS and PFS endpoints in all participants as well as in participants whose tumours expressed PD-L1. Secondary endpoints comprise objective Response risk (ORR), response time(DOR),disease control rate(DCR), and health.

TNBC is a type of breast cancer which is particularly aggressive. Within the first five years after diagnosis, it has a high rate of recurrence. The triple-negative applies to negative tests of all three hormone receptors, oestrogen receptor, progesterone receptor and receptor of human epidermal growth factor 2 (HER2). Most cancer treatments target one or more of these hormone receptors, and this lack of all three makes the
cancer-targeting difficult for therapies. TNBC affects 15-20percent of women with breast cancer.
https://www.biospace.com/article/merck-s-checkpoint-inhibitor-keytruda-hits-another-mark-in-breast-cancer/



Eli Lilly Announces Topline Results for the Solanezumab from the Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) Study

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Eli Lilly Announces Topline Results for the Solanezumab from the Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) Study

Feb 10, 2020: Eli Lilly and Company announced that the analysis performed by the Washington University School of Medicine in the Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) Study showed that the solanezumab did not meet the primary endpoint. Additional analyses of secondary endpoints and biomarkers are ongoing by the Washington University and Lilly.

Data will be presented at the Advances in Alzheimer’s and Parkinson’s Therapies (AAT-AD/PD™) Focus Meeting in April of 2020. At this time, Lilly does not plan to chase a submission for solanezumab in people with dominantly inherited Alzheimer’s disease (DIAD), also known as autosomal dominant Alzheimer’s disease, based on results of the primary endpoint. This outcome does not force the ongoing solanezumab Anti-Amyloid Treatment in the Asymptomatic Alzheimer’s (A4) Study.

The experiment on the DIAN-TU platform is a randomized, double-blind, placebo-controlled, Phase 2/3 test. The goal is to study possible disease-modifying treatments in individuals at risk for or with Alzheimer’s disease dominantly inherited, which is caused by unusual gene mutations. This started as a two-year study of biomarker goal involvement and progressed into a Phase 2/3 analysis of registration with a primary measure of cognitive performance and a total of four years of treatment. The primary efficiency analysis included 50 solanezumab and 40 placebo participants. The minimum four-year treatment period was finished by 36 solanezumab and 32 placebo participants. The initial dose for the study was 400 mg every 4 weeks. A late change to the analysis raised the dose, which resulted in approximately 25% of the total doses being administered at 1600 mg.

The DIAN-TU Study was established in 2010 and funded by Lilly, Roche and Genentech, National Institutes of Health, and other donors and is the first disease prevention trial in order to test investigational Alzheimer’s disease compounds with different mechanisms of action from two pharmaceutical companies.

Solanezumab is an investigational anti-amyloid monoclonal antibody tested in the Asymptomatic Alzheimer’s (A4) analysis of preclinical Alzheimer’s disease in Anti-Amyloid Therapy. The A4 Study is a clinical trial that studies solanezumab in older people who have signs of amyloid in their brains, but do not show memory loss symptoms.

The DIAN-Multivariate Cognitive Endpoint (DIAN MCE) is composed of Wechsler Memory ScaleRevised Logical Memory Delayed Recall, Cogstate International Shopping List Test, Wechsler Adult Intelligence ScaleRevised Digit Symbol Substitution Test, and Mini-Mental State Analysis.https://investor.lilly.com/news-releases/news-release-details/lilly-announces-topline-results-solanezumab-dominantly-inherited

Novartis announces MET inhibitor capmatinib, the first potential treatment for the METex14 mutated advanced non-small cell lung cancer, granted priority FDA review

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Novartis announces MET inhibitor capmatinib, the first potential treatment for the METex14 mutated advanced non-small cell lung cancer, granted priority FDA review

Feb 11, 2020:  Novartis announced that the U.S.FDA accepted and granted Priority Review to capmatinib’s (INC280) NDA. Capmatinib is a MET inhibitor being evaluated as a treatment for the  first-line and formerly treated patients with locally advanced or metastatic MET exon 14 skipping (METex14) mutated non-small cell lung cancer (NSCLC). If accepted, capmatinib will be the first therapy to specially target METex14 mutated advanced lung cancer, a type of the lung cancer with a particularly poor prognosis.

Priority Review is granted to therapies that the FDA determines have the potential in order to provide considerable improvements in the treatment, diagnosis or prevention of the serious conditions. This designation shortens the FDA review period following the approval of the NDA to six months compared to ten months under Standard Review. Previously, Novartis granted Breakthrough Therapy designation for capmatinib.There are currently no approved therapies that especially target METex14 mutated advanced NSCLC. NSCLC accounts for about 85% of the lung cancer diagnoses. METex14 mutations occur in 3-4% of newly diagnosed superior NSCLC casesand is a recognized oncogenic driver.

The NDA submission for capmatinib is maintained by results from the GEOMETRY mono-1 Phase II study, which established an overall response rate of 67.9% (95% CI, 47.6 – 84.1) and 40.6% (95% CI, 28.9 – 53.1) among treatment-naïve and formerly treated patients, respectively, based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.1. The study also demonstrated that the capmatinib provided durable responses among all the patients: median duration of response was 11.14 months (95% CI, 5.55 – NE) in the treatment-naïve patients and 9.72 months (95% CI, 5.55 – 12.98) in formerly treated patients.

All results were based on the independent appraisal by the BIRC, and all tumor CT scans were evaluated in parallel by two radiologistsin order to confirm the response. The most common treatment-related adverse events (AE) (≥ 10% all grades) across all the cohorts (N=334), were peripheral edema (42%), nausea (33%), creatinine increase (20%), vomiting (19%), fatigue (14%), decreased appetite (13%) and also diarrhea (11%).

Capmatinib (INC280) is an investigational, oral, active, and selective MET inhibitor that 
Incyte Corporation licensed to Novartis in 2009. Under the Agreement, Incyte has given 
exclusive development and marketing rights to capmatinib and certain back up compounds 
worldwide to Novartis in all indications.
https://www.novartis.com/news/media-releases/novartis-announces-met-inhibitor-capmatinib-inc280-first-potential-treatment-metex14-mutated-advanced-non-small-cell-lung-cancer-granted-priority-fda-review

Bayer and Nuvisan generate new research unit in Berlin

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Bayer and Nuvisan generate new research unit in Berlin

Feb 11, 2020: Bayer AG announced that it entered into a definitive agreement to transfer a large part of its Berlin-based small molecule research unit to Nuvisan, an international service provider for clinical studies, laboratory services and contract manufacturing with several sites and clinics in Germany and France.

The strategic partnership will arrange the foundation for a brand-new research entity to be established by the Nuvisan in Berlin. At the same time, it will support Bayer’s improved focus on the flexibility and productivity of its R&D operating model.

Financial terms were not disclosed.
The Berlin-based research unit with around 400 workplaces comprises of a fully operational team specialized in the small molecule research.

The research center comes with capabilities and capacities across the entire drug discovery value chain, including Lead Discovery, Medicinal Chemistry, Pharmacology, Drug Metabolism & Pharmacokinetics, Investigational Toxicology, and Animal Management.

Bayer will maintain significant research activities in the Berlin, which remains the headquarters for the company’s Pharmaceuticals Division and one of its major global research sites.
Bayer and Nuvisan will work together in the coming years to build the new research center. The transaction is expected to seal mid of 2020 subject to successful completion of the consultation process with the employee representatives and the preparations for succession the research activities.


The Nuvisan group creates sales of approx. € 55 million with currently about 420 highly qualified employees. It originated from the LAB GmbH over 40 years ago and has been operating under the name NUVISAN with its headquarters in Neu-Ulm since 2010.

The Nuvisan group as the CRO and CDMO has six sites in Germany and France (Neu-Ulm, Berlin, Grafing, Gauting, Waltrop and Sophia-Antipolis) in addition to the monitoring activities with offices in Argentina, Peru and Brazil as well as in the USA.
https://media.bayer.com/baynews/baynews.nsf/id/Bayer-and-Nuvisan-create-new-research-unit-in-Berlin

U.S. FDA granted fast track designation for Nanobiotix for the investigation of first-in-class nbtxr3 in head and neck cancer

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U.S. FDA granted fast track designation for Nanobiotix for the investigation of first-in-class nbtxr3 in head and neck cancer

Feb 10, 2020: NANOBIOTIX , a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer announced that the U.S. FDA  has granted Fast Track designation for the investigation of NBTXR3 activated by the radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced head and neck squamous cell cancer who are not appropriate for platinum-based chemotherapy.

Fast Track is a process designed in order to facilitate the development and accelerate the review of drugs for the serious conditions and that have the potential to address unmet medical needs. The purpose is to speed up the availability of new treatment options for the patients.
The products that receives Fast Track designation is eligible for :
 • More frequent meetings with the FDA to examine the drug’s development plan and make sure about the collection of appropriate data needed to support drug approval.
 • More numerous written communication from the FDA about such things as the design of the proposed clinical trials and the use of biomarkers.
 • Eligibility for the Accelerated Approval and Priority Review, if relevant criteria are met.
• Rolling Review, which means that the drug company can submit completed sections of the NDA for review by the FDA, rather than waiting until the entire NDA is completed before the application can be reviewed

 NBTXR3 NBTXR3 is a first-in-class product designed in order to destroy tumors through physical cell death when activated by the radiotherapy. It has a high degree of biocompatibility, requires one single administration before the first radiotherapy treatment session, and has the capability to fit into current worldwide radiotherapy radiation therapy standards of the care.
https://www.nanobiotix.com/wp-content/uploads/2020/02/PR_ENGLISH-Fast-Track-Designation.pdf

Astex Pharmaceuticals announces U.S. Food and Drug Administration (FDA) acceptance for the review of NDA for the combination oral hypomethylating agent cedazuridine and decitabine for the treatment of MDS and CMML

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Astex Pharmaceuticals announces U.S. Food and Drug Administration (FDA) acceptance for the review of NDA for the combination oral hypomethylating agent cedazuridine and decitabine for the treatment of MDS and CMML

Feb 11, 2020: Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Japan  announced that the U.S. FDA has accepted for Priority Review its NDA for oral C-DEC (cedazuridine and decitabine) as a treatment for adult patients with previously untreated intermediate- and high-risk MDS including CMML.

The NDA submission is based on the data from the ASCERTAIN phase 3 study which evaluated the 5-day decitabine exposure equivalence of oral C-DEC and IV decitabine.

The FDA grants Priority Review to the applications for drugs that, if approved, would provide significant improvements in the safety and efficacy of the treatment, diagnosis or prevention of serious conditions. The Priority Review designation means the FDA’s goal is to take action on an NDA application within six months. Oral C-DEC (investigational compound) not currently approved in any country.

 About C-DEC (Cedazuridine 100 mg and Decitabine 35 mg) Fixed-Dose CombinationC-DEC is a novel, orally administered fixed dose combination of the cedazuridine, an inhibitor of cytidine deaminase with the anti-cancer DNA hypomethylating agent, decitabine.

It inhibit cytidine deaminase in the gut and the liver, C-DEC is designed to permit for oral delivery of the approved DNA hypomethylating agent, decitabine, at exposures which follow exposures achieved with the approved intravenous form of the decitabine administered over 5 days.


C-DEC has been evaluated in the phase 1/2 pharmacokinetics-guided dose escalation and dose confirmation study in the patients with MDS and CMML see https://www.clinicaltrials.gov NCT02103478) and also pivotal phase 3 study (ASCERTAIN)see https://www.clinicaltrials.gov NCT03306264) conducted at investigator sites in the US and Canada and designed in order to confirm the results from the phase 1/2 study.

The phase 3 study is now being comprehensive to include patients with acute myeloid leukemia (AML) unsuitable to receive intensive induction chemotherapy.

Astex announced that C-DEC had received orphan drug designation in September 2019 for the treatment of MDS and CMML from the U.S. FDA. The concept of using cedazuridine is to block the action of cytidine deaminase is also being evaluated in the low dose formulation of cedazuridine and decitabine for the treatment of lower risk MDS (see   https://www.clinicaltrials.gov NCT03502668).

About Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML): Myelodysplastic syndromes are a heterogeneous group of the hematopoietic stem cell disorders characterized by the dysplastic changes in myeloid, erythroid, and megakaryocytic progenitor cells, and associated with cytopenias distressing one or more of the three lineages.  U.S. incidence of the MDS is estimated to be 10,000 cases per year, even though the condition is thought to be under-diagnosed.
https://astx.com/astex-pharmaceuticals-announces-u-s-food-and-drug-administration-fda-acceptance-for-review-of-an-nda-for-the-combination-oral-hypomethylating-agent-cedazuridine-and-decitabine-astx727-or-oral-c-de/

U.S. FDA Acceptance of New Drug Application for FibroGen’s Roxadustat for the Treatment of Anemia of Chronic Kidney Disease

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U.S. FDA Acceptance of New Drug Application for FibroGen’s Roxadustat for the Treatment of Anemia of Chronic Kidney Disease

Feb. 11, 2020: FibroGen announced that the U.S.FDA has completed its filing review of its New Drug Application (NDA) for roxadustat for the treatment of anemia of chronic kidney disease (CKD), both in non-dialysis-dependent (NDD) and dialysis-dependent (DD) patients. The application will be measured filed on Feb. 18, 2020. The FDA has set a Prescription Drug User Fee Act (PDUFA) date of the December 20, 2020. 

“The FDA’s acceptance of the roxadustat NDA  is the critical step towards as long as a new treatment option in the United States for the chronic kidney disease patients suffering from anemia, a severe and often life-threatening disease,” said Enrique Conterno, Chief Executive Officer, FibroGen.

The filing of the roxadustat NDA triggers  $50 million milestone payment from AstraZeneca to FibroGen. Roxadustat is the first small molecule hypoxia-inducible factor prolyl hydroxylase (HIFPH)orally administered inhibitor approved by the FDA for the treatment of CKD anaemia.The submission is supported by positive results from the global Phase 3 program encircling more than 8,000 patients.

Roxadustat is currently approved in China for the treatment of anemia in the patients with CKD, regardless of whether they necessitate dialysis, and in Japan for the treatment of dialysis patients with anemia of the CKD.FibroGen’s partner Astellas also plans to file an application for marketing authorisation with the European Medicines Agency in the first half of 2020.

About Anemia Associated with CKD: Anemia results from the reduced oxygen-carrying capacity of the blood, in which patients usually have insufficient red blood cells and/or low levels of the hemoglobin, a protein in red blood cells that carries oxygen to cells all the way through the body. CKD anemia, which can be associated with the increased risk of hospitalization, cardiovascular complications, and death, also recurrently causes significant fatigue, cognitive dysfunction, and reduced quality of the life. Severe anemia is common in patients with the CKD, cancer, myelodysplastic syndromes (MDS), inflammatory diseases, and other severe illnesses.
https://fda.einnews.com/pr_news/509481763/fibrogen-announces-u-s-fda-acceptance-of-new-drug-application-for-roxadustat-for-the-treatment-of-anemia-of-chronic-kidney-diseases

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