Attention Biotech Investors: Mark Your Calendar For May PDUFA Dates

Zinger Key Points
  • The PDUFA calendar for May is relatively light, with six investigational therapies awaiting regulatory nod
  • Phathom looks ahead to its maiden approval, while Verrica is hoping to see no further delays following multiple disappointments

The month of April was a mixed one for regulatory approvals. The Food and Drug Administration delayed approvals for at least three treatment candidates. New molecular entity, or NME, approvals, a measure of innovation in drug research, tapered to merely one for the month.

BioXcel Therapeutics, Inc. BTAI was among the fortunate ones, as the biopharma snagged approval for its Igalmi as a treatment option for agitation associated with neurological disorders.

Bristol-Myers Squibb Company's BMY heart disease drug mavacamten, which came into its stable following the Myokardia acquisition, cleared the regulatory hurdle. The FDA also gave its nod for the label expansion of Supernus Pharmaceutical, Inc.'s SUPN Qelbree to be used as a treatment option for attention deficit hyperactivity syndrome in adults.

Here are the key PDUFA dates scheduled for May:

Can Phathom Snag Its Maiden Nod?

Company: Phathom Pharmaceuticals, Inc. PHAT
Type of Application: new drug applications
Candidate: Vonoprazan
Indication: bacterial infection
Date: May 3

Phathom, a clinical-state biopharma, submitted two NDAs to the FDA in September 2021, seeking approvals of two vonoprazan combinations, one with amoxicillin and another with clarithromycin and amoxicillin, as treatment options for Helicobacter pylori infection in adults.

The current standard-of-care therapies, according to the company, now have reduced impact for eradicating the bacterial infection in the U.S. It sees the two combo treatments as offering two new therapeutic options providing superior eradication rates as compared to the standard-of-care of lansoprazole-based triple therapy.

Contingent on the FDA approving the two combinations, the company expects a launch in the U.S. in the second half of 2022.

Pfizer-Myovant Hope For Myfembree Label Expansion Despite FDA Warning Of Deficiency In Application

Company: Pfizer, Inc. PFE and Myovant Sciences, Inc. MYOV
Type of Application: supplemental NDA
Candidate: Myfembree
Indication: endometriosis related severe pain
Date: May 6

Myfembree, co-developed and commercialized by Pfizer and Myovant, is being evaluated for the management of moderate-to-severe pain associated with endometriosis. It is a convenient one pill, once-a-day treatment option.

The companies announced last month that the FDA communicated to them certain deficiencies in the NDA that preclude discussion of labeling and/or post-marketing

In May 2021, the FDA approved the treatment for managing heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months.

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Can Third Time Be The Charm For Verrica?

Company: Verrica Pharmaceuticals Inc. VRCA
Type of Application: NDA
Candidate: VP-102
Indication: viral skin infection
Date: May 24

VP-102, or cantharidin 0.7% topical solution, is being evaluated as a potential treatment option for molluscum contagiosum and common warts. Molluscum, according to Verrica, is a highly contagious viral skin infection affecting approximately six million people, primarily children, in the U.S.

Verrica's original NDA submitted in late 2019 was rejected by the FDA, and following a resubmission by the company, a new PDUFA goal date was announced. The company faced another setback when the drug regulator extended the review period by three months.

Following the extended review, the FDA issued a second complete response letter in September 2021, citing deficiencies at a facility of a contract manufacturer. Verrica's second resubmission was accepted for review in mid-December.

Bristol-Myers Squibb Awaits Approval For Immunotherapy Combo In Esophageal Cancer

Company: Bristol-Myers Squibb
Type of Application: supplemental biologic license application
Candidate: Opdivo and Yervoy
Indication: esophageal squamous cell carcinoma
Date: May 28

The combination of Bristol-Myers Squibb's cancer immunotherapies Opdivo and Yervoy are being evaluated as a first-line treatment for adults with advanced, metastatic esophageal squamous cell carcinoma.

According to statistics quoted by the company, about 19,000 people were diagnosed with esophageal cancer in the U.S. in 2020 and 15,000 died. The Opdivo-Yervoy combo will likely improve upon outcomes with the current standard-of-care, the company said.

Can Amicus Take Its Rare Genetic Disease Treatment Past The Finish Line?

Company: Amicus Therapeutics, Inc. FOLD
Type of Application: NDA
Candidate: miglustat for AT-GAA
Indication: Pompe disease
Date: May 29

AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, administered in conjunction with miglustat. It is being evaluated for an inherited lysosomal disorder. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time.

Pompe disease ranges from a rapidly fatal infantile form with significant impacts on heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

Eton Hopes to Earn Milestone Payment From Epilepsy Drug Approval

Company: Azurity Pharma
Type of Application: NDA
Candidate: ET-105 (lamotrigine for oral suspension)
Indication: epilepsy
Date: May

Azurity Pharma acquired lamotrigine from Eton Pharmaceuticals, Inc. ETON in February 2021, and it has submitted to the FDA results of the product's human factor study in the fourth quarter of 2021. Following this, the agency assigned a PDUFA goal date of May. Eton stands to receive $5 million in milestone payment from Azurity upon the approval and launch of lamotrigine.

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