Attention Biotech Investors: Mark Your Calendar For August PDUFA Dates

The month of July was disappointing from the perspective of Food and Drug Administration decisions, as the agency handed down mostly negative verdicts.

Provention Bio, Inc. PRVB, Incyte Corporation INCY and Iterum Therapeutics plc ITRM were among the companies, which faced outright rejections.

ChemoCentryx, Inc. CCXI, however, was asked to wait for three more months, as the FDA extended the review period for avacopan as a treatment option for anti-neutrophil cytoplasmic autoantibody-associated vasculitis. The stock reacted positively on a relief rally. Pfizer Inc. PFE said the FDA notified that the PDUFA dates for abrocitinib in atopic dermatitis and label expansion for Xeljanz in active ankylosing spondylitis were unlikely to be met.

Among the fortunate ones were Merck & Co., Inc. MRK, which received approval for V114, an investigational 15-valent pneumococcal conjugate vaccine, and also for Keytruda-chemo combo in first-line breast cancer setting.

Albireo Pharma, Inc. ALBO obtained the nod for odevixibat for treating pruritus in patients with progressive familial intrahepatic cholestasis, a rare inherited progressive liver disease.

Four new molecular entities were approved during the month, taking the total for thus far in the year to 31.

Here are the key PDUFA dates for the month of August:

Can Eton Score FDA Win For Seizure Drug?

• Company: Eton Pharmaceuticals, Inc.ETON
• Type of Application: NDA
• Candidate: topiramate oral solution
• Indication: seizures
• Date: Aug. 6

Topiramate is one of Eton's three neurology-focused oral liquid product candidates that have been submitted to the FDA.

Eton is seeking approval of topiramate oral solution as a treatment option for three indications – as a monotherapy for partial-onset or primary general tonic-clonic seizures in patients two years age and older, an adjunctive treatment of partial-onset seizures, including seizures associated with Lennox-Gastaut syndrome in patients two years of age and older, and as preventative treatment of migraine in patients 12 years of age and older.

In recent months, Eton has had mixed fortune with respect to regulatory approvals. In late May, the FDA handed down a complete response letter for its NDA for dehydrated alcohol injection for the treatment of methanol poisoning. The regulatory agency, however, cleared the company's Rezipres, or ephedrine hydrochloride injection, for the treatment of hypotension occurring in the setting of anesthesia.

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Jazz Hopes For Expanding the Label of Sleep Disorder Drug

• Company: Jazz Pharmaceuticals plc JAZZ
• Type of Application: supplemental NDA
• Candidate: Xywav (JZP-258)
• Indication:
• Date: Aug. 12

Xywav is calcium, magnesium, potassium and sodium oxybates oral solution. It was first approved in July 2020 for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy.

The company is now seeking to expand the label of Xywav to include idiopathic hypersomnia in adults as the second inducation. The regulatory application was accepted for priority review on April 12.

Sesen Bio Seeks Approval For Bladder Cancer Drug

• Company: Sesen Bio, Inc. SESN
• Type of Application: biologic license application
• Candidate: vicinium
• Indication: Bladder cancer
• Date: Aug. 18

Vicineum is a locally administered fusion protein. It's Sesen Bio's lead product candidate that is being investigated for the treatment of high-risk, BCG-unresponsive non-muscle invasive bladder cancer.

The FDA accepted the application for priority review on Feb. 16.

Sesen Bio sees peak revenue of $1 billion to $3 billion globally, with $400 million to $900 million coming from the U.S.

Canaccord Genuity analyst John Newman said Vicineum displays an overall better safety and clinical profile compared to Keytruda, which should lead to FDA approval.

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Sanofi Looks to Get Its Pompe Disease Treatment Past The Finish Line After 3-Month Delay

• Company: Sanofi SNY
• Type of Application: BLA
• Candidate: avalglucosidase alfa
• Indication: Pompe disease
• Date: Aug. 18

Avalglucosidase alfa, an enzyme replacement therapy, is being evaluated for the treatment of patients with Pompe disease, a rare degenerative muscle disorder that can impact an individual's ability to move and breathe. It affects an estimated 3,500 people in the U.S. and can manifest at any age from infancy to late adulthood.

The original PDUFA date of May 18 was extended by three months.

Axsome Awaits Approval For Its Potentially First Commercial Product

• Company: Axsome Therapeutics, Inc. AXSM
• Type of Application: NDA
• Candidate: Dextromethorphan-bupropion (AXS-05)
• Indication: major depressive disorder
• Date: Aug. 22

AXS-05, chemically dextromethorphan-bupropion, is an oral investigational drug with multimodal activity under development for the treatment of major depressive disorder and other central nervous system disorders.

The FDA accepted the NDA for priority review on April 26. If approved, Axsome expects to commercially launch the drug in the fourth quarter.

‘Go or no-go' For Cara's Korsuva?

• Company: Cara Therapeutics, Inc. CARA and Vifor Pharma
• Type of Application: Pruritus In hemodialysis Patients
• Candidate: Korsuva injection
• Indication: NDA
• Date: Aug. 23

Cara and partner Vifor Pharma announced FDA acceptance of the NDA for Korsuva on March 8. Korsuva, or difelikefalin, solution for injection is being investigated for the treatment of moderate-to-severe pruritus in hemodialysis patients.

Cara is entitled to 50:50 share of U.S. profit in Fresenius clinics and 60:40 share in non-Fresenius clinics, Needham analyst Joseph Stringer said in a note. Approval in the U.S. will pave the way for a $50 million equity investment and up to $240 million in U.S. sales milestones, he added.

Can Merck-Eisai's Combo Therapy Cross The FDA Hurdle?

• Company: Merck & Eisai Co., Ltd. ESALY
• Type of Application: sBLA and sNDA
• Candidate: Keytruda and Lenvima
• Indication: kidney cancer
• Date: Aug. 25 and 26

The FDA accepted and granted priority review for the applications seeking approvals for the combination of Merck's Keytruda and Eisai's Lenvima for the first-line treatment of patients with advanced renal cell carcinoma.