Attention Biotech Investors: Mark Your Calendar For March PDUFA Dates

The Food and Drug Administration approval calendar was light in February. Nevertheless, the month saw regulatory nods accorded to some breakthrough therapies.

Agios Pharmaceuticals, Inc.'s AGIO Pyrukynd (mitapivat) was approved for the treatment of hemolytic anemia in people with pyruvate kinase deficiency. Hemolysis refers to the destruction of red blood cells.

Pyrukynd is a "new molecular entity," a term that refers to a drug containing an active moiety that hasn't been previously approved by the FDA. NME approval, therefore, is considered a measure of innovation in drug research.

Another NME that passed the FDA hurdle was Sanofi's SNY Enjaymo for the treatment of hemolysis in adults with cold agglutinin disease.

Reata Pharmaceuticals, Inc. RETA, as expected, received a complete response letter for its new drug application for bardoxolone methyl as a treatment option for Alport syndrome. An Adcom that met in early December voted against recommending approval of the drug.

PDUFA dates are key "binary or make-or-break catalysts" for biopharma companies. These are dates by which the drug regulator is required to announce its verdict on regulatory applications filed with it, following either a standard review period or priority review.

Here are the key PDUFA dates scheduled for February.

Click here to access Benzinga's FDA Calendar

Bristol-Myers Squibb Seeks Nod For Skin Cancer Combo Therapy

• Company: Bristol-Myers Squibb Company BMY
• Type of Application: biologic license application
• Candidate: relatlimab and Opdivo
• Indication: melanoma
• Date: March 19

Relatlimab is an antibody, and it's being tested along with Bristol-Myers Squibb's cancer immunotherapy Opdivo, in a fixed-dose combo, as a single infusion for treating adult and pediatric patients with melanoma that has spread and cannot be removed by surgery.

The FDA accepted the company's regulatory application for priority review in September 2021.

Marinus Positive On Seizure Drug Approval

• Company: Marinus Pharmaceuticals, Inc. MRNS
• Type of Application: NDA
• Candidate: ganaxolone
• Indication: seizures
• Date: March 20

Marinus' ganaxolone is an investigational product being developed in intravenous and oral formulations, and it shows both anti-seizure and anti-anxiety activity due to its effects on synaptic and extrasynaptic GABAa receptors.

The FDA accepted for priority review the company's NDA, seeking approval for ganaxolone as a treatment option for seizures associated with CDKL5 deficiency disorder, in September. This is a rare genetic epilepsy.

Marinus management is confident of approval coming through and is expecting no REMS or black box warning on the label, SVB Leerink analyst Marc Goodman said in a mid-February note. The company is looking ahead to a launch timeline of July following the 90-day Drug Enforcement Administration scheduling process.

The company recently announced a delay in the readout from the Phase 3 study of intravenous ganaxolone in refractory status epilepticus, citing omicron impact and an interruption of clinical supply material associated with the drug.

Will Zogenix Gain Label Expansion For Seizure Drug?

• Company: Zogenix, Inc. ZGNX
• Type of Application: supplemental NDA
• Candidate: Fintepla (fenfluramine)
• Indication: seizures
• Date: March 25

Fintepla has already been approved in the U.S. and Europe for treating seizures associated with Dravet syndrome. Zogenix has now applied for expanding the label to include seizures associated with Lennox-Gastaut syndrome, which is rare and difficult to treat childhood developmental and epileptic encephalopathy.

Zogenix has recently announced an agreement to be acquired by Belgian biopharma UCB.

TG Therapeutics Could See A Pushback In Blood Cancer Combo Therapy Review

• Company: TG Therapeutics, Inc. TGTX
• Type of Application: BLA
• Candidate: ublituximab, in combination with Ukoniq (umralisib)
• Indication: chronic lymphocytic leukemia and small lymphocytic lymphoma
• Date: March 25

Ukoniq is currently indicated for the treatment of adult patients with relapsed or refractory marginal zone lymphoma, who have received at least one prior anti-CD20-based regimen, and for the treatment of adult patients with relapsed or refractory follicular lymphoma, who have received at least three prior lines of systemic therapy.

TG Therapeutics' application seeks approval for ublituximab, an antibody treatment, along with Ukoniq for VLL and SLL. It was accepted for review in May 2021. The company said in November the FDA has notified it regarding a proposed meeting of the Oncologic Drugs Advisory Committee in connection with its review. With still no word on a confirmed date for the Adcom meeting, a decision by the stipulated date is unlikely.

Related Link: Moderna Rallies After Q4 Earnings: What 2 Analysts Have To Say About COVID-19 Stock

Bristol-Myers Squibb Awaits Third Nod For Reblozyl

• Company: Bristol-Myers Squibb
• Type of Application: sBLA
• Candidate: Reblozyl (luspatercept)
• Indication: anemia in adults with non-transfusion dependent beta thalassemia
• Date: March 27

Reblozyl is an erythroid maturation agent that promotes late-stage red blood cell maturation in animal models. It's being jointly developed by Bristol-Myers Squibb and & Merck & Company, Inc. MRK in the U.S. Merck received rights to the treatment through its acquisition of Acceleron.

Can Merck Snag Another Approval For Keytruda?

• Company: Merck
• Type of Application: sBLA
• Candidate: Keytruda
• Indication: endometrial carcinoma
• Date: March 28

Merck has filed for getting another regulatory approval for Keytruda, this time as a single agent for the treatment of patients with advanced endometrial carcinoma that is microsatellite instability-high or mismatch repair deficient.

Lipocine Aims To Convert Tlando Tentative Approval To Full Approval

• Company: Lipocine Inc. LPCN/Antares Pharma, Inc. ATRS
• Type of Application: NDA
• Candidate: Tlando (testosterone undecanoate)
• Indication: treating adult males with hypogonadism
• Date: March 28

Tlando is an oral prodrug of testosterone containing testosterone undecanoate, and has received tentative approval from the FDA in December for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism. The final approval was delayed, as the exclusivity period previously granted to Clarus Therapeutics' rival product Jatenzo expires only on March 27.

Lipocine has an exclusive licensing agreement with Antares for marketing Tlando in the U.S.

Akebia Hopes For Positive Tidings On Anemia Drug

• Company: Akebia Therapeutics, Inc. AKBA
• Type of Application: NDA
• Candidate: Vadadustat
• Indication: anemia in chronic kidney disease patients.
• Date: March 29

Akebia and Japanese partner Otsuka Holdings Co., Ltd. OTSKY announced last June that the FDA accepted the NDA filing for vadadustat as a treatment option for anemia due to chronic kidney disease in both adult patients on dialysis and adult patients not on dialysis.

Adcom Calendar

FDA's Peripheral and Central Nervous System Drugs Advisory Committee is scheduled to meet on March 30 to discuss Amylyx Pharmaceuticals, Inc.'s AMLX NDA for sodium phenylbutyrate/taurursodiol powder for oral suspension, for the treatment of amyotrophic lateral sclerosis.