After a fairly robust 2021, the new year started on a mixed note as far as regulatory decisions are concerned.
About three new molecular entities, or NMEs, were approved during the month, including Immunocore Holdings plc's (NASDAQ:IMCR) Kimmtrak for certain patients with advanced eye melanoma and Pfizer, Inc.'s (NYSE:PFE) atopic dermatitis treatment Cibinqo.
NME approval is an important indicator to measure innovation in drug research. NME approvals totaled 50 in 2021, almost equaling the numbers of the previous year.
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)-Sanofi (NASDAQ:SNY), however, failed to get the nod for expanding the Libtayo label to include cervical cancer. Pfizer also faced rejection for its pediatric growth hormone deficiency treatment Somatrogon.
A PDUFA date is the date/period by which the Food and Drug Administration is required to announce its verdict on a sponsor company's regulatory application. This will determine the fate of millions of dollars invested in developing the treatment from the preclinical-stage to clinical testing. Therefore, it is a key binary catalyst for a stock.
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Here are the key PDUFA dates scheduled for February:
Agios Seeks Approval For Drug To Treat Rare, Inherited Disease That Leads to Accelerated Destruction Of RBCs
- Company: Agios Pharmaceuticals, Inc. (NASDAQ:AGIO)
- Type of Application: new drug application
- Candidate: Mitapivat
- Indication: pyruvate kinase deficiency
- Date: Feb. 17
Mitapivat is Agios' most advanced drug candidate, and it is a pyruvate kinase R activator that is currently being evaluated for the treatment of three distinct hemolytic anemias. Pyruvate kinase deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells.
Agios' application for Mitapivat was accepted for priority review by the FDA on Aug. 7.
Reata Hopes For A Miracle After Adcom Recommends Against Approving Kidney Disease Drug
- Company: Reata Pharmaceuticals, Inc. (NASDAQ:RETA)
- Type of Application: NDA
- Candidate: bardoxolone methyl
- Indication: Alport syndrome
- Date: Feb. 25
Bardoxolone is an investigational, once-daily, orally administered drug that is being evaluated as a treatment option for Alport syndrome is a rare, genetic form of chronic kidney disease caused by mutations in the genes.
The FDA's Cardiovascular And Renal Drugs Advisory Committee, which met on Dec. 8 to discuss the NDA, voted "no" on the question of whether the provided evidence demonstrated that bardoxolone is effective in slowing the progression of CKD in patients with Alport syndrome and that its benefits outweigh its risks.
Can Amryt's Rare Skin Disorder Drug Clear FDA Hurdle After 3-Month Delay
- Company: Amryt Pharma plc (NASDAQ:AMYT)
- Type of Application: NDA
- Candidate: Filsuvez (Oleogel-S10)
- Indication: epidermolysis bullosa
- Date: Feb. 28
Filsuvez is a potential treatment for the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa, a rare genetic skin disorder affecting young children and adults for which there is currently no approved treatment.
The initial PDUFA date of Nov. 30 was extended by three months to give the FDA time to review additional information provided by the company upon the regulator's request.
'No' or ‘No-Go' For Legend Biotech's T-Cell Therapy For Blood Cancer?
- Company: Johnson & Johnson (JNJ)/Legend Biotech Corporation (NASDAQ:LEGN)
- Type of Application: biologic license application
- Candidate: ciltacabtagene autoleucel (Cilta-cel)
- Indication: multiple myeloma
- Date: Feb. 28
Cilta-cel is a B-cell maturation antigen CAR-T therapy that is being investigated for the treatment of adults with relapsed and/or refractory multiple myeloma. Legend Biotech entered into an exclusive worldwide license and collaboration agreement with J&J's Janssen unit in 2017 to develop and commercialize cilta-cel.
Legend Biotech began the rolling submission of the application in December 2020 and the application was granted priority review in May 2021, with the PDUFA date of Nov. 29. In late October, the FDA communicated a 3-month delay in the review.
Will Gilead's Long-Acting HIV Drug Clear The FDA Hurdle?
- Company: Gilead Sciences, Inc. (NASDAQ:GILD)
- Type of Application: NDA
- Candidate: Lenacapavir
- Indication: HIV-1 infection
- Date: Feb. 28
Gilead is seeking approval for lenacapavir, an investigational, long-acting HIV-1 capsid inhibitor, for the treatment of HIV-1 infection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection.
CTI BioPharma's Rare Blood Cancer Drug Awaits FDA Nod
- Company: CTI BioPharma Corp. (NASDAQ:CTIC)
- Type of Application: NDA
- Candidate: Pacritinib
- Indication: myelofibrosis patients with severe thrombocytopenia
- Date: Feb. 28
The FDA accepted the regulatory application for priority review on June 1 and announced a PDUFA date of Nov. 30. The decision date was later extended by three months to Feb. 28.
Pacrotinib, if approved, is expected to address the unmet medical need of myelofibrosis patients with severe thrombocytopenia.
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Adcom Calendar
FDA's Oncologic Drugs Advisory Committee is scheduled to meet on Feb. 10 to discuss BLA for sintilimab injection, submitted by Eli Lilly & Company's (NYSE:LLY) partner Innovent Biologics.
The proposed indication for this product is in combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of patients with Stage IIIB, IIIC, or Stage IV non-squamous non-small cell lung cancer with no epidermal growth factor receptor or anaplastic lymphoma kinase genomic tumor aberrations.
A joint meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and the Drug Safety and Risk Management Advisory Committee is tentatively scheduled for Feb. 15, to discuss Avenue Therapeutics, Inc.'s (NASDAQ:ATXI) NDA for intravenous tramadol for the U.S. market.
The NDA for the pain drug was shot down twice by the FDA and subsequently, the company filed a formal dispute resolution request with the FDA's Office of New Drugs in August. The OND has sought additional advisory committee inputs to resolve the dispute.
