From time to time, the U.S. FDA requests additional data from companies who have submitted an application for a new drug or biologic. What’s a bit unusual is every single PDUFA date this week had that happen.
From time to time, the U.S. Food and Drug Administration (FDA) requests additional data from companies who have submitted an application for a new drug or biologic, and because of the significance and amount of the data, require more time to analyze it. In which case, they typically add three months to the PDUFA date. What’s a bit unusual is every single PDUFA date this week had that happen. Here’s a look.
Incyte’s Ruxolitinib Cream for Atopic Dermatitis and Ruxolitinib for GVHD
Incyte Corporation has a target action date of September 21, 2021, for its New Drug Application (NDA) for ruxolitinib cream for atopic dermatitis (AD). The original date was three months earlier, but after the company submitted additional data per the agency’s request, the agency decided they needed additional time to analyze it.
Ruxolitinib cream is a proprietary formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib designed for topical application. It is in Phase III development for adolescents and adults with AD (TRuE-AD) and vitiligo (TRuE-V).
In June, the company presented 52-week safety and efficacy data from the two Phase III TRuE-AD trials at the Revolutionizing Atopic Dermatitis (RAD) virtual symposium. The data was the basis of the NDA. They had announced the data in May, which hit the primary and key secondary endpoints, including patient reported outcomes.
Incyte also has a target action date of September 22 for its supplemental New Drug Application (sNDA) for Jakafi (ruxolitinib) for adults and pediatric patients 12 years and older with steroid-refractory chronic graft-versus-host disease (GVHD). The FDA also extended the PDUFA date by three months for time to analyze data they had requested. The data for the sNDA came from the Phase III REACH3 trial comparing Jakafi with best available therapy (BAT) in this indication and age group.
At the company’s second-quarter financial report on August 3, company Chief Executive Officer Herve Hoppenot, said, “We continue to achieve clinical development success across our portfolio with positive outcomes from multiple programs, including topline results from ruxolitinib cream’s pivotal trials in vitiligo (TRuE-V), 52-week safety and efficacy data of ruxolitinib cream in atopic dermatitis (TRuE-AD), Phase II data of parsaclisib in autoimmune hemolytic anemia (AIHA) and the achievement of bioequivalence with once-daily (QD) ruxolitinib.”
Verrica’s VP-102 for Molluscum Contagiosum
Verrica Pharmaceuticals has a target action date of September 23 for its NDA for VP-102. It also had a PDUFA date three months earlier but was extended by three months because data submitted after an agency request was viewed as a Major Amendment and required more time for full analysis.
Molluscum is a highly contagious viral skin disease that affects about six million people, mostly children, in the U.S. alone. It is the result of a pox virus and presents as distinctive raised, skin-toned-to-pink-colored lesions that can be painful, inflamed, itchy and lead to bacterial infections.
VP-102 is a proprietary drug-device combination that contains a GMP-controlled formulation of cantharidin delivered by way of a single-use applicator. If approved, it would be marketed under the brand name Ycanth.
At the company’s second-quarter financial report on August 10, Ted White, Verrica’s President and Chief Executive Officer, stated, “This quarter, we continued to ramp up commercial preparations for the potential FDA approval of VP-102, our lead product candidate for the treatment of molluscum contagiosum, including strengthening our senior leadership team and ensuring patient access to VP-102 through productive dialogue with medical providers and payors. With a strong financial position, and a PDUFA goal date of September 23, 2021, we continue to invest in our commercial capabilities and, if approved, we look forward to the opportunity to launch VP-102 in the fourth quarter of 2021.”
Ascendis Pharma’s Lonapegsomatropin for Pediatric Growth Hormone Deficiency
Ascendis Pharma had a target action date of September 25 for its Biologics License Application (BLA) for lonapegsomatropin for pediatric growth hormone deficiency (GHD). As with every other company in this week’s FDA Action Alert, it originally had a PDUFA date three months earlier, but it was extended three months after the company submitted data to meet an agency request.
In this case, however, the BLA was approved on August 25. The drug will be marketed under the brand name Skytrofa and is indicated for pediatric patients one year and older who weigh at least 11.5 kg and have growth failure from inadequate secretion of endogenous growth hormone (GH). The drug is a once-weekly injection. The approval also includes the Skytrofa Auto-Injector and cartridges.
“Today’s approval represents an important new choice for children with GHD and their families, who will now have a once-weekly treatment option,” said Paul Thornton, clinical investigator and pediatric endocrinologist in Forth Worth, Texas. “In the pivotal head-to-head clinical trial, once-weekly Skytrofa demonstrated higher annualized height velocity at week 52 compared to somatropin.”
