Most Food and Drug Administration decisions scheduled for February were positive, and more importantly five new molecular entities passed FDA muster.
NME approvals for the month included EMD Serono's Tepmetko for treating adult patients with certain types of non-small cell lung cancer, Regeneron Pharmaceuticals Inc's REGN cholesterol drug Evkeeza and Sarepta Therapeutics Inc's SRPT Duchenne muscular dystrophy drug Amondys 45.
Mallinckrodt PLC MNKKQ is forced to wait, as the FDA deferred action on its biologics license application for StrataGraft for deep partial-thickness burns. The delay is due to the FDA's inability to complete site inspection.
Here are the key decisions due for March:
KemPharm's Novel Formulation Of ADHD Drug Awaits Clearance
Company: KemPharm Inc KMPH & Aquestive Therapeutics Inc AQST
Type of Application: New drug application
Candidate: KP415
Indication: Attention deficit hyperactivity disorder
Date: March 2
Following a late-cycle communication meeting with the FDA, the company said in early December the agency did not raise any substantive issues related to safety and efficacy at that point in its review.
KemPharm's KP415 consists of serdexmethylphenidate, the company's prodrug of d-methylphenidate, co-formulated with immediate release d-methylphenidate. KP415, according to the company, is designed to address unmet needs with the most widely-prescribed methylphenidate ADHD treatments. Specifically, it brings about earlier onset of action and longer duration of therapy, while also avoiding unnecessary spikes in d-MPH concentrations associated with adverse events.
KemPharm is collaborating with Aquestive Therapeutics for an oral film dosage of KP415.
Can Gilead Get Label Expansion For Cell-Therapy to Treat Lymphoma?
Company: Gilead Sciences, Inc. GILD
Type of Application: Supplemental biologics license application
Candidate: Axicabtagene ciloleucel (Yescarta)
Indication: Follicular and marginal zone lymphoma
Date: March 5
Yescarta is a chimeric antigen receptor, or CAR, T-cell therapy developed by Kite Pharma, which was acquired by Gilead in 2017.
CAR T-cell therapy is a technology where the patient's T lymphocytes are collected and transducted with a gene that encodes for a CAR to direct T cells against cancer cells. These genetically modified autologous T cells are expanded in the lab and then reinfused into the patient.
Yescarta was first approved in 2017 for treating large B-cell lymphoma in patients, who have not responded to or have relapsed after at least two other kinds of treatment.
Gilead is now seeking expansion in label to include the indications of relapsed or refractory follicular lymphoma and marginal zone lymphoma, after two or more prior lines of systemic therapy.
Fibrogen-AstraZeneca Hope To Get Anemia Drug Past The Finish Line
Company: FibroGen Inc FGEN & AstraZeneca plc AZN
Type of Application: New drug application
Candidate: Roxadustat
Indication: Anemia of chronic kidney disease
Date: March 20
Roxadustat is an oral medicine and belongs to a class of medicines called HIF-PH inhibitors that promote erythropoiesis, or RBC production. It has already been approved in China, Japan and Chile for the treatment of anemia of chronic kidney disease in adult patients both on dialysis and not on dialysis.
The NDA pending before the FDA seeks 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 collaborating with AstraZeneca on the development and commercialization of roxadustat for the treatment of anemia in the U.S., other markets in the Americas, China, Australia, New Zealand and Southeast Asia.
The original PDUFA date of Dec. 20 was extended by three months. Fibrogen said at that time it is submitting additional analyses of existing roxadustat clinical data, which require an extension of the original PDUFA date.
Related Link: Johnson & Johnson Secures Emergency Authorization For Its Single-Shot COVID-19 Vaccine
Kiniksa Seeks Label Expansion For In-licensed Drug
Company: Kiniksa Pharmaceuticals Ltd KNSA
Type of Application: Supplemental biologics license application
Candidate: Rilonacept
Indication: Recurrent pericarditis
Date: March 21
The sBLA for rilonacept was accepted for priority review by the FDA in November, with a PDUFA goal 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 REGN and is approved by the FDA under the brand name Arcalyst for the treatment of cryopyrin-associated periodic syndromes, specifically familial cold autoinflammatory syndrome and Muckle-Wells Syndrome, and DIRA. Kiniksa licensed rilonacept from Regeneron in 2017.
If approved by the FDA for recurrent pericarditis, Kiniksa will take responsibility for sales and distribution of rilonacept for all the approved indications in the U.S. and evenly split profits with Regeneron.
Recurrent pericarditis is swelling and irritation of the thin, sac-like tissue surrounding the heart and can cause sharp chest pain.
In its fourth quarter earnings release on Feb. 23, Kiniksa said it is preparing for the commercial launch of rilonacept in recurrent pericarditis in the first half of 2021.
Pacira's Key Non-Opioid Pain Drug On Track to Get Label Expansion to Include Pediatric Population
Company: Pacira Biosciences Inc PCRX
Type of Application: Supplemental new drug application
Candidate: Exparel
Indication: Post-surgical analgesia in children
Date: March 22 (expected)
Exparel, or bupivacaine liposome injectable suspension, is currently indicated for single-dose infiltration in adults to produce postsurgical local analgesia and as an interscalene brachial plexus nerve block to produce postsurgical regional analgesia.
The company is now seeking expansion of the Exparel label to include single-dose infiltration to provide postsurgical analgesia in children, aged six and over.
Exparel accounted for over 97% of Pacira's total revenues of $429.65 million in 2020.
Zealand Joins The Fray With Anticipated Approval of Dasiglucagon Rescue Pen to Treat Very Low Blood Sugar Levels
Company: Zealand Pharma A S ADR ZEAL
Type of Application: New drug application
Candidate: Dasiglucagon
Indication: Rescue medication for low blood sugar levels in diabetic patients
Date: March 27
The NDA for the dasiglucagon HypoPal rescue pen was accepted for review in May of last year.
Zealand's ready-to-use dasiglucagon HypoPal rescue pen is designed to offer diabetes patients fast and effective treatment for severe hypoglycemia, the company said. Three Phase 3 trials in adults and pediatrics showed a median time to blood glucose recovery of only 10 minutes following injection of 0.6 mg of dasiglucagon, it added.
The company said it remains on track for the potential launch of the pen early this year.
The market for diabetic rescue medication is getting crowded. Xeris Pharmaceuticals Inc's XERS Last July, the Gvoke HypoPen was launched in the U.S. In July 2019, Eli Lilly And Co's LLY nasal glucagon powder was approved.
Go Or No-Go For Bristol-Myers Squibb & Bluebird Bio's T-Cell Therapy For Multiple Myeloma
Company: bluebird bio Inc BLUE & Bristol-Myers Squibb Co BMY
Type of Application: Biologics license application
Candidate: Idecabtagene vicleucel, or ide-cel/bb2121
Indication: Multiple myeloma
Date: March 27
FDA accepted the BLA for priority review in September. Idecabtagene vicleucel, or ide-cel, is being evaluated for adult patients with multiple myeloma, who have received at least three prior therapies.
It is being co-developed by Bristol-Myers Squibb and bluebird bio, who aim to win FDA approval by the end of this month. The approval is one of the required remaining milestones related to Bristol-Myers Squibb's buyout of Celgene.
Ide-cel is classed as an investigational B-cell maturation, antigen-directed, CAR T-cell immunotherapy.
Another Nod In The Cards For Merck's Wonder Cancer Drug?
Company: Merck & Co., Inc. MRK
Type of Application: Supplemental new drug application
Candidate: Keytruda KN-522
Indication: Breast cancer
Date: March 29
The FDA is set to rule on Merck's hugely successful cancer immunotherapy Keytruda. This time around, it will be in combination with chemotherapy for the treatment of patients with certain types of breast cancer. Specifically, it targets locally recurrent, unresectable or metastatic triple-negative breast cancer whose tumors express PD-L1.
Aveo Seeks FDA Approval For Kidney Cancer Drug
Company: AVEO Pharmaceuticals, Inc. AVEO
Type of Application: New drug application
Candidate: Tivozanib
Indication: Renal cell cancer
Date: March 31
Tivozanib is being evaluated as a treatment option for relapsed or refractory renal cell carcinoma.
The FDA accepted the application last June for standard review. The NDA submission is based on Aveo's pivotal Phase 3 study, TIVO-3, comparing tivozanib to Bayer AG's BAYRY sorafenib, the current standard-of-care, in third- and fourth-line renal cell cancer.
It is formally described as Aveo's next-generation vascular endothelial growth factor receptor tyrosine kinase inhibitor.
Adcom Meetings
FDA's Arthritis Advisory Committee in conjunction with the Drug Safety and Risk Management Advisory Committee is scheduled to meet March 24-25 to discuss Pfizer Inc.'s PFE BLA for a treatment for osteoarthritis in adult patients. The tanezumab subcutaneous injection is aimed at patients for whom use of other analgesics is ineffective or not appropriate.
Related Link: The Week Ahead In Biotech (Feb. 28-March 6): KemPharm, Gilead FDA Decisions and More Earnings
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