Attention Biotech Investors: Mark Your Calendar For April PDUFA Dates

Biopharma stocks are sensitive to a key binary event called the PDUFA date, the date by which the Food and Drug Administration is required to give its verdict on a regulatory application filed with it.

The month of March saw several approvals coming through, while outright rejection and postponements were few and far between. Three new molecular entities, or NMEs, were approved during the month. NMEs are drugs that contain active moieties that haven't been previously approved by the FDA. It's, therefore, an indicator of innovation in drug research.

Marinus Pharmaceuticals, Inc.'s MRNS Ztalmy, indicated to treat seizures associated with a rare genetic condition known as CDKL-5 deficiency disorder (CDD), was among the NMEs that scored win the FDA in March. H.C. Wainwright analyst Douglas Tsao expects peak U.S. sales potential of $92 million for Ztalmy, given CDD is a relatively small market.

Other NMEs approved during the month included Bristol-Myers Squibb Company's BMY melanoma combo therapy Opdualag and Novartis AG's NVS Pluvicto for treating a certain type of advanced, castration-resistant prostate cancer.

On the flip side, Akebia Therapeutics, Inc.'s AKBA vadadustat was rejected by the FDA as a treatment option for anemia in chronic kidney disease patients.

Here are the key PDUFA dates scheduled for April.

Click here to access Benzinga's FDA Calendar

Can BioXCel Push Psychiatric Disorder-Associated Agitation Drug Past Finish Line?

• Company: BioXcel Therapeutics, Inc. BTAI
• Type of Application: new drug application
• Candidate: dexmedetomidine (BXCL501)
• Indication: agitation associated with schizophrenia and bipolar disorders I and II.
• Date: April 5

BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine for the treatment of agitation associated with neuropsychiatric disorders. The original PDUFA date of Jan. 5, 2022 was pushed back by three months, with BoXcel communicating that the FDA needed three more months to review data pertaining to analyses of clinical data submitted by the company upon the regulator's information request.

Regeneron Awaits Approval Of Antibody Cocktail For Prophylactic Treatment Of COVID-19

• Company: Regeneron Pharmaceuticals, Inc. REGN
• Type of Application: BLA
• Candidate: REGEN-COV
• Indication: prophylactic treatment of COVID-19
• Date: April 13

Regeneron's REGEN-COV is a cocktail of two monoclonal antibodies, namely casirivimab and imdevimab, that's designed to block infectivity of SARS-CoV-2 using the company's proprietary VeloImmune and VelociSuite technologies. The biopharma has a collaboration agreement with Roche Holding AG RHHBY for increasing global supply.

Alnylam Hopes To Get Clearance For Second RNAi Therapeutic For ATTR Amyloidosis

• Company: Alnylam Pharmaceuticals, Inc. ALNY
• Type of Application: NDA
• Candidate: Vutrisiran
• Indication: polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) polyneuropathy
• Date: April 14

Vutrisiran is an investigational RNAi therapeutic being evaluated for treating hATTR in adults. hATTR is an inherited, progressively debilitating and fatal disease caused by mutations in the TTR gene, which will cause the build up of amyloid protein in organs and tissues. Vutrisiran is a second-generation, subcutaneous formulation of Alnylam's Onpattro, which was approved for the same indication. Onpattro is administered intravenously.

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No or no-go For Amneal's Avastin Biosimilar

• Company: Amneal Pharmaceuticals, Inc. AMRX
• Type of Application: BLA
• Candidate: Bevacizumab
• Indication: colorectal cancer
• Date: April 17

Amneal's Bevacizumab regulatory application was accepted for standard review pursuant to Section 351(k) pathway of the Public Health Service Act, to the Biosimilar User Fee Act. The biosimilar was developed in collaboration with mAbxience, a Spain-based biotechnology company. Bevacizumab is the biosimilar version of Roche's Genentech unit's Avastin. Upon approval, the company expects to market it under the proprietary name Alymsys.

Bevacizumab is a vascular endothelial growth factor inhibitor. Amneal is seeking approval for Bevacizumab for the treatment of metastatic colorectal cancer. According to IQVI, U.S. annual sales for Bevacizumab for the 12 months ended April 2021 were approximately $2.8 billion.

Bristol-Myers Squibb Hopes For No Further Heartache On Heart Disease Drug

• Company: Bristol-Myers Squibb Company BMY & LianBio LIAN
• Type of Application: NDA
• Candidate: mavacamten
• Indication: symptomatic obstructive hypertrophic cardiomyopathy (oHCM)
• Date: April 28

Mavacamten is a cardiac myosin inhibitor that demonstrated clinically meaningful improvements in symptoms, functional status, and quality of life in symptomatic oHCM patients in a pivotal trial. The investigational therapy came into Bristol-Myers Squibb's stable through its Myokardia acquisition. Myokardia and LianBio initially collaborated to develop mavacamten.

HCM is a chronic, progressive disease characterized by the abnormal thickening of heart muscle, which makes it difficult for the heart to pump blood. It affects one in every 500 persons and can lead to cardiac dysfunction and other debilitating symptoms.

In November, the company communicated an extension to the review period for mavacamten, and gave a new PDUFA date of April 28.

Can Supernus Snag Label Expansion For ADHD Drug?

• Company: Supernus Pharmaceuticals, Inc. SUPN
• Type of Application: supplemental NDA
• Candidate: Qelbree (viloxazine extended-release capsules)
• Indication: attention deficit hyperactivity disorder
• Date: April 29

Qelbree received its first approval in April 2021 for treating ADHD in pediatric patients, ages six to 17 years. The company now expects to make the treatment available to adult patients, who according to the company number around 10 million in the U.S.

Chinese Biopharma Hutchmed Knocks FDA Door For Approval Of Neuroendocrine Tumor Treatment Candidate

• Company: HUTCHMED (China) Limited HCM
• Type of Application: NDA
• Candidate: surufatinib
• Indication: pancreatic and extra-pancreatic neuroendocrine tumors (NET)
• Date: April 30

Hutchmed's surufatinib is marketed in China under the brand name Sulanda. Surufatinib is evaluated as a treatment option for NET, which forms in cells interacting with the nervous system or in glands that produce hormones. It originates mostly in the gut and lungs and can be benign or malignant.

Will Axsome Add A Drug To Crowded Migraine Market?

• Company: Axsome Therapeutics, Inc. AXSM
• Type of Application: NDA
• Candidate: meloxicam-rizatriptan (AXS-007)
• Indication: acute migraine
• Date: April 30

AXS-07 (MoSEIC meloxicam-rizatriptan) is an oral, rapidly absorbed, multi-mechanistic, investigational medicine for migraine. According to CDC, over 37 million Americans suffer from migraine.

AXS-007, if approved, will be joining a crowded field, comprising AbbVie, Inc. ABBV, Eli Lilly & Company LLY and Biohaven Holding Company Limited BHVN.

Coherus, Chinese Partner Seek Approval For Nasopharyngeal Cancer Treatment

• Company: Coherus BioSciences, Inc. CHRS
• Type of Application: BLA
• Candidate: Toripalimab
• Indication: nasopharyngeal caner
• Date: April 30

Coherus is developing toripalimab, an anti PD-1 monoclonal antibody, along with China's Junshi Biosciences. The investigational treatment is being evaluated in combination with gemcitabine and cisplatin for the first-line treatment for patients with advanced recurrent or metastatic nasopharyngeal carcinoma and toripalimab monotherapy for the second-line or above treatment of recurrent or metastatic NPC after platinum-containing chemotherapy.

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Adcom Calendar

FDA's Oncologic Drugs Advisory Committee is scheduled to meet on April 22 to discuss TG Therapeutics, Inc.'s TGTX sNDA for Ukoniq (umbralisib) tablets, and BLA for ublituximab injection. The proposed indication for these two products is in combination for the treatment of adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma.

The committee will also discuss the existing umbralisib indications in patients with relapsed or refractory follicular lymphoma and marginal zone lymphoma approved under accelerated approval regulations.