FDA Action Alert: Urovant, Athenex, scPharmaceuticals and Vertex

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The U.S. Food and Drug Administration (FDA) is plenty busy with COVID-19 vaccine Emergency Use Authorizations (EUAs) this month, but they’re also wrapping up the year with a few PDUFA dates for other therapies. Here’s a look.

Urovant Sciences’ Vibegron for Overactive Bladder

Urovant Sciences has a target action date of December 26, 2020, for its New Drug Application for once-daily 75 mg vibegron for patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency.

On May 14, the company presented data from the vibegron EMPOWUR 52-week extension study and data by age groups from the 12-week placebo-controlled EMPOWUR study at the 2020 American Urological Association Annual Meeting. The data supported the potential benefits of the drug for OAB in patients with urge urinary incontinence (UUI), urgency, and urinary frequency. The drug is a once-daily, beta-3 adrenergic agonist.

David Staskin, principal investigator of EMPOWUR and a urologist with St. Elizabeth’s Medical Center and associate professor of Urology at Tufts University School of Medicine, said at the time, “The results of the EMPOWUR study over the 52-week period demonstrated the sustained benefits of vibegron. Vibegron could be a potentially important and differentiated new oral treatment, if approved by the FDA, for patients suffering with OAB.”

Athenex’ Klisyri For Actinic Keratosis

Athenex had a target action date of December 30 for its NDA for Klisyri (tirbanibulin) ointment for treatment of actinic keratosis (AK). It was approved on December 15, 2020.

The submission included data from two positive pivotal Phase III trials. The studies achieved the primary endpoint of 100% clearance of AK lesions on Day 57 within the face or scalp treatment areas.

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. and Europe, including Russia.

Actinic Keratosis is a common skin condition caused by UV light damage. It results in patches of thick, scaly or crusty skin. It can progress to squamous cell carcinoma if untreated. It is the most common pre-cancerous condition and affects more than 55 people in the U.S.

“The FDA approval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. “Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. We are extremely proud of our team’s excellent execution. Approval demonstrates our ability to execute upon the entirety of the drug development and registration process. We are excited to partner with Almirall to bring this first-in-class microtubule inhibitor to patients with actinic keratosis in the U.S.”

scPharmaceuticals’ Furoscix for Worsening Heart Failure Due to Congestion

scPharmaceuticals had a target action date of December 30 for its resubmitted NDA for Furoscix for treatment of worsening heart failure due to congestion. The FDA completed a class 2 response in July 2020. Furoscix is a proprietary furosemide solution formulated to a neutral pH so it can be infused subcutaneously for fluid overload (congestion) in patients with heart failure.

On December 3, 2020, the FDA issued a complete response letter (CRL) for the NDA. In the rejection, the agency indicated they need to conduct pre-approval inspections at two of scPharmaceuticals’ third-party manufacturing facilities that they couldn’t do because of travel restrictions related to the COVID-19 pandemic. The FDA also had questions regarding testing, labeling, and features of the combination product that were not related to the drug constituent. The FDA also said there were deficiencies at the third-party facility where scPharmaceuticals’ off-the-shelf alcohol swabs are manufactured. The company has requested a Type A meeting with the FDA to discuss the issues in the CRL.

“While we are disappointed that these on-site inspections, and other issues raised in the CRL, will not be resolved by our previously granted December 30, 2020 PDUFA date, we are committed to working with our manufacturing partners and responding to the agency’s concerns as expeditiously as possible,” said John Tucker, scPharmaceuticals’ chief executive officer. “We continue to believe that Furoscix can play a significant role in preventing heart failure hospital admissions and readmissions due to fluid overload by intervening with this novel therapy at home.”

Vertex’s Three sNDA’s for Trikafta, Symdeko and Kalydeco for Cystic Fibrosis

Vertex Pharmaceuticals has a target action date of December 30 for three supplemental New Drug Applications (sNDAs) for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor). The sNDA are for expansion of the labels for all three drugs to include additional rare CFTR mutations. This would allow people with cystic fibrosis (CF) who were not previously eligible for the drug the opportunity to use them. They also suggest that for people with CF who are currently using Kalydeco may be eligible for Symdecko or Trikafta and people currently eligible for Symdeko may become eligible for Trikafta.

CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. It is caused by a defective and/or missing CFTR protein caused by mutations in the CFTR gene. The most common mutation is having at least one F508del.

The sNDAs are based on in vitro data from a validated cell assay model demonstrating that many rare mutations in the CFTR gene respond to one or more of the three drugs beyond the mutations that they are currently approved for. Previous studies from the model, as well as Phase III clinical data, have resulted in almost 30 additional ultra-rare and rare mutations for Kalydeco and Symdeko, as well as the first ever FDA approval based on in vitro data for a Kalydeco label expansion in patients with residual function CFTR mutations.

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