FDA Action Alert: BioSpecifics and Endo, Eagle Pharma, and Verrica
It’s a relatively slow month ahead for July in terms of PDUFA dates for the U.S. Food and Drug Administration (FDA). However, there are several dates pending throughout the month. Here’s a look at three for the next two weeks.
BioSpecifics and Endo’s Xiaflex for Cellulite
BioSpecifics Technologies Corporation and Endo International’s Xiaflex (collagenase clostridium histolyticum) has a target action date of July 6, 2020, for a new Biologics License Application (BLA) for the treatment of cellulite in the buttocks. Xiaflex is approved for Dupuytren’s Contracture and Peyronie’s disease. It is also being developed for adhesive capsulitis and plantar fibromatosis.
The BLA was built on the results of two Phase III trials, RELEASE-1 and RELEASE-2, which evaluated 845 women aged 18 years or older in the U.S. with moderate-to-severe cellulite. The studies showed the drug demonstrated a clinically meaningful and statistically significant improvement compared to placebo.
Eagle Pharma’s Ryanodex for Exertional Heat Stroke
Eagle Pharmaceuticals has a target action date of July 8 for its resubmitted New Drug Application (NDA) for Ryanodex (dantrolene sodium for injectable suspension) for exertional heat stroke. The company resubmitted the NDA in January 2020. It originally received a Complete Response Letter (CRL) from the FDA in July 2017, indicating that the agency required an additional clinical trial before it would approve the drug for that indication. The resubmitted NDA included data collected from 41 patients treated for exertional heat stroke during the Hajj pilgrimage.
There are no approved pharmacological treatments for exertional heat stroke. Treatment is typically cooling the body using water immersion, ice packs, or water misting, and supportive measures such as intravenous fluids and respiratory support.
Ryanodex is indicated for the treatment of malignant hyperthermia in conjunction with appropriate supportive measures and for preventing malignant hyperthermia in patients at high risk. Malignant hyperthermia is a severe reaction in response to particular medications used during general anesthesia. Symptoms include muscle rigidity, high fever, and a fast heart rate.
Exertional heat stroke is a severe form of heat-related illness, marked by core body temperate of 104º F (40º C) or greater, with significant neurological dysfunction, including sudden changes in behavior, seizures or coma.
Verrica Pharmaceuticals’ VP-102 for Molluscum Contagiosum
Verrica Pharmaceuticals has a target action date of July 13 for its NDA for VP-102 for molluscum contagiosum. The drug is a potent first-in-class topical therapy. Molluscum contagiosum is a skin lesion caused by a poxvirus. IfF approved, the company plans to market the drug under the brand name Ycanth.
However, on June 29, the company received a letter from the FDA stating there were deficiencies that preclude discussion of labeling and post-marketing requirements at that time. It also stated that the notification doesn’t reflect a final decision.
The company indicated, “The FDA’s letter does not identify any specific items. But, the Company notes that information requests from the FDA during the NDA review have focused on CMC aspects of the drug-device combination. Verricia’s ability to address these CMC-related requests, however, was significantly impacted in large part by the COVID-19 pandemic.”
The requests included data related to a possible safety issue with the applicator if the instructions weren’t properly followed. The company responded by adding an additional user feature into the applicator, but that addition affected human factors testing, which required additional supportive stability data on the fully assembled device. “The Company believes that both its long-term and registration stability data with the ampule, and the as-submitted applicator, support significant shelf life and stability for VP-102.”
There are currently no indications if the PDUFA date is being rescheduled or still holds at July 13. Verrica does say it “believes that the positive results from its two double-blind Phase III trials (CAMP-1 and CAMP-2) that evaluated the safety and efficacy of VP-102 compared to placebo in patients two years of age and older diagnosed with molluscum indicate that VP-102 remains viable for FDA approval.”