Attention Biotech Investors: Mark Your Calendar For March PDUFA Dates

Most of the Food and Drug Administration decisions scheduled for February have been positive, and more importantly, five new molecular entities have passed FDA screening.
This month's NME approvals included EMD Serono's Tepmetko for the treatment of adult patients with certain types of non-small cell lung cancer, Regeneron Pharmaceuticals Inc.'s (NASDAQ: REGN) cholesterol drug Evkeeza, and Duchenne muscular dystrophy drug Amondys 45 from Sarepta Therapeutics Inc. (NASDAQ: SRPT).
Mallinckrodt PLC (OTC: MNKKQ) has to wait as FDA postponed action on its biologics license application for StrataGraft due to deep burns with partial thickness. The delay is due to the FDA's inability to complete the on-site inspection.
Here are the top decisions due in March:
KemPharm's novel formulation of ADHD drugs is awaiting approval
Company: KemPharm Inc (NASDAQ: KMPH) and Aquestive Therapeutics Inc (NASDAQ: AQST)
Type of application: New drug application
Candidate: KP415
Indication: attention deficit hyperactivity disorder
Date: March 2nd
After a late-cycle communication meeting with the FDA, the company announced in early December that the agency had not raised any material safety or efficacy-related questions in its review at that time.
KemPharms KP415 is composed of Serdexmethylphenidate, the Company's prodrug for d-methylphenidate, formulated with immediate-release d-methylphenidate. KP415 is designed to meet unmet needs with the most widely prescribed methylphenidate ADHD treatments, according to the company. In particular, it causes an earlier onset of action and a longer duration of therapy, while avoiding unnecessary spikes in d-MPH concentrations associated with adverse events.
KemPharm partnered with Aquestive Therapeutics for an oral film dose of KP415.
Can Gilead get an extension to its cell therapy label for the treatment of lymphoma?
Company: Gilead Sciences, Inc. (NASDAQ: GILD)
Type of application: Additional license application for biologics
Candidate: Axicabtagene ciloleucel (Yescarta)
Indication: Follicular and peripheral zone lymphoma
Date: March 5th
Yescarta is a chimeric antigen receptor or CAR-T cell therapy developed by Kite Pharma that was acquired by Gilead in 2017.
CAR T cell therapy is a technology in which the patient's T lymphocytes are collected and transduced with a gene that encodes a CAR to target T cells against cancer cells. These genetically modified autologous T cells are expanded in the laboratory and then re-infused into the patient.
Yescarta was first approved in 2017 for the treatment of large B-cell lymphoma in patients who have failed or have relapsed to at least two other types of treatment.
Gilead is now seeking to expand the marker to include the indications of relapsed or refractory follicular lymphoma and marginal zone lymphoma after two or more previous systemic lines of therapy.
Fibrogen-AstraZeneca hopes to bring anemia drug across the finish line
Companies: FibroGen Inc (NASDAQ: FGEN) and AstraZeneca plc (NASDAQ: AZN)
Type of application: New drug application
Candidate: Roxadustat
Indication: anemia in chronic kidney disease
Date: March 20th
Roxadustat is an oral medicine and belongs to a class of medicines called HIF-PH inhibitors that help increase erythropoiesis, or RBC production. It has already been approved in China, Japan and Chile for the treatment of anemia associated with chronic kidney disease in adult patients on both dialysis and non-dialysis.
The FDA-pending NDA is filing for approval of Roxadustat for the treatment of anemia in chronic kidney disease in both non-dialysis-dependent and dialysis-dependent patients.
Fibrogen, the sponsor of the application, is working with AstraZeneca to develop and commercialize roxadustat for the treatment of anemia in the US, other markets in the Americas, China, Australia, New Zealand and Southeast Asia.
The original December 20th PDUFA date has been extended by three months. Fibrogen said at this point that it is providing additional analysis of existing roxadustat clinical data that requires an extension of the original PDUFA date.
Related link: Johnson & Johnson Secures Emergency Approval for its Single-Shot COVID-19 Vaccine
Kiniksa is seeking label expansion for a licensed drug
Company: Kiniksa Pharmaceuticals Ltd (NASDAQ: KNSA)
Type of application: Additional license application for biologics
Candidate: Rilonacept
Indication: recurrent pericarditis
Date: March 21
The sBLA for rilonacept was approved for priority review by the FDA in November with a PDUFA target date of March 21. Rilonacept is a weekly subcutaneously injected recombinant dimeric fusion protein that blocks interleukin-1 alpha and interlukin-1 beta signaling.
It was discovered by Regeneron Pharmaceuticals Inc (NASDAQ: REGN) and is FDA approved under the brand name Arcalyst for the treatment of cryopyrin-associated periodic syndromes, specifically familial cold autoinflammatory syndrome and Muckle-Wells syndrome, as well as DIRA. Kiniksa licensed Rilonacept from Regeneron in 2017.
If Kiniksa is FDA cleared for recurrent pericarditis, Kiniksa will take responsibility for the sale and distribution of Rilonacept for all approved indications in the US and will split profits equally with Regeneron.
Recurrent pericarditis is swelling and irritation of the thin, baggy tissue that surrounds the heart and can cause sharp chest pain.
In its fourth quarter earnings release on February 23, Kiniksa said it is preparing to launch rilonacept for recurrent pericarditis commercial in the first half of 2021.
Pacira's premier non-opioid pain reliever on track to extend label extension to the pediatric population
Company: Pacira Biosciences Inc (NASDAQ: PCRX)
Type of application: Complementary new drug application
Candidate: Exparel
Indication: Postoperative analgesia in children
Date: March 22nd (expected)
Exparel or Bupivacaine Liposome Injectable Suspension is currently indicated for single dose infiltration in adults to produce postoperative local analgesia and as an interscalene nerve block of the brachial plexus to produce postoperative regional analgesia.
The company is now aiming to expand the Exparel label to include a single-dose infiltration to enable postoperative analgesia in children aged six and over.
Exparel accounted for over 97% of Pacira's total revenue of $ 429.65 million in 2020.
Zealand joins the battle with the expected approval of the Dasiglucagon Rescue Pen for the treatment of very low blood sugar levels
Company: Zealand Pharma A S ADR (NASDAQ: ZEAL)
Type of application: New drug application
Candidate: Dasiglucagon
Indication: Rescue drugs against low blood sugar levels in diabetics
Date: March 27th
The NDA for the Dasiglucagon HypoPal Rescue Pen was approved for review last May.
The ready-to-use Dasiglucagon HypoPal Rescue Pen in Zealand is designed to provide diabetes patients with quick and effective treatment for severe hypoglycemia, the company said. Three phase 3 studies in adults and in pediatrics showed a median time to glucose recovery of only 10 minutes after injection of 0.6 mg dasiglucagon.
The company said it was still on track for the pen's possible launch earlier this year.
The market for diabetic rescue drugs is getting denser. Xeris Pharmaceuticals Inc. (NASDAQ: XERS) The Gvoke HypoPen was launched in the United States last July. In July 2019, the nasal glucagon powder was approved by Eli Lilly And Co (NYSE: LLY).
Go-or-no-go for T-cell therapy from Bristol-Myers Squibb & Bluebird Bio for multiple myeloma
Companies: Bluebird Bio Inc. (NASDAQ: BLUE) and Bristol-Myers Squibb Co. (NYSE: BMY)
Type of application: Biologics license application
Candidate: Idecabtagene vicleucel or ide-cel / bb2121
Indication: multiple myeloma
Date: March 27th
The FDA accepted the BLA for priority review in September. Idecabtagene vicleucel or ide-cel is being studied for adult patients with multiple myeloma who have previously received at least three therapies.
It is being jointly developed by Bristol-Myers Squibb and Bluebird Bio, who plan to receive FDA approval by the end of this month. The approval is one of the required remaining milestones in connection with the acquisition of Celgene by Bristol-Myers Squibb.
Ide-cel is classified as an antigen-directed CAR T-cell immunotherapy to study B-cell maturation.
Another nod in the cards for Merck's miracle cancer drug?
Company: Merck & Co., Inc. (NYSE: MRK)
Type of application: Complementary new drug application
Candidate: Keytruda KN-522
Indication: breast cancer
Date: March 29th
The FDA will decide on Merck's hugely successful Keytruda cancer immunotherapy. This time it is used in combination with chemotherapy to treat patients with certain types of breast cancer. In particular, it targets locally recurring, unresectable or metastatic triple negative breast cancer whose tumors express PD-L1.
Aveo applies for FDA approval for kidney cancer drugs
Company: AVEO Pharmaceuticals, Inc. (NASDAQ: AVEO)
Type of application: New drug application
Candidate: tivozanib
Indication: renal cell cancer
Date: March 31st
Tivozanib is currently being evaluated as a treatment option for relapsed or refractory renal cell carcinoma.
The FDA approved the application for standard testing last June. The NDA filing is based on Aveo's pivotal Phase 3 study TIVO-3, which compares tivozanib with Bayer AG's (OTC: BAYRY) sorafenib, the current standard of care for third and fourth lineage renal cell cancer.
It is officially described as a next-generation vascular endothelial growth factor receptor tyrosine kinase inhibitor by Aveo.
Adcom meetings
The FDA's Arthritis Advisory Committee, in conjunction with the Drug Safety and Risk Management Advisory Committee, is expected to meet March 24-25 to discuss Pfizer Inc.'s (NYSE: PFE) BLA for the treatment of osteoarthritis in adult patients. The subcutaneous injection of tanezumab is intended for patients in whom the use of other analgesics is ineffective or unsuitable.
Related Link: The Week Ahead of Us in Biotech (February 28 - March 6): KemPharm, Gilead FDA Decisions and More Revenue
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The Week Before Biotech (February 28 - March 6): KemPharm, Gilead FDA decisions and more revenue
The Daily Biotech Pulse: Adcom Test Awaits COVID-19 Vaccine From J&J, Lilly To Deliver Additional Doses Of Antibody Therapy To U.S., Regulatory Backlash For Tricida
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