Amryt Pharma Advances Regulatory Applications for Filsuvez

Amryt Pharma has completed its rolling submission of an application to the U.S. Food and Drug Administration seeking the approval of Filsuvez (Oleogel-S10), a topical gel to heal skin wounds in people with junctional (JEB) and dystrophic epidermolysis bullosa (DEB).

The new drug application (NDA) includes a request for priority review, which can quicken the review process to six months following submission acceptance. The rolling submission of the NDA began June 29.

In parallel, the European Medicines Agency (EMA) has validated an application seeking a similar approval in the European Union. This means the EMA deems Amryt Pharma‘s marketing authorization application (MAA) sufficiently complete to launch the formal review process. 

An opinion from the Committee for Medicinal Products for Human Use (CHMP), an arm of the EMA, is expected in about seven months, excluding any days needed for the company to answer questions from the CHMP.

“Our NDA submission to the FDA marks another important milestone for Amryt as we progress our lead development candidate Oleogel-S10 with the regulatory authorities in both the US and Europe,” Joe Wiley, CEO of Amryt, said in a press release.

“Today’s news also represents a potentially important advancement for patients and families living with this rare and distressing disorder. We will continue to work closely with the respective regulatory authorities with the hope of bringing Oleogel-S10 to patients as soon as possible,” Wiley stated in another press release. 

The therapy first was approved in 2016 in the European Union under the brand name Episalvan to treat partial-thickness skin wounds, in which the top layers of the skin have been lost due to burns or skin grafts. Clinical trials were initiated to investigate whether Filsuvez is effective for EB wounds.

Filsuvez is a birch bark extract rich in betulin, which is thought to promote wound healing by controlling the inflammatory response and enhance the migration of keratinocytes — the main cell type of the outer layer of skin (epidermis).

The MAA is supported by results of the EASE study, a Phase 3 clinical trial (NCT03068780) evaluating Filsuvez in 156 children and 67 adults with JEB, DEB or Kindler syndrome, all rare skin disorders characterized by fragile skin prone to injury and blisters. 

The trial met its primary goal of accelerated healing of wounds in patients treated with Filsuvez. A statistically significant 41.3% of those treated with the medicine had first complete target wound closure within 45 days, compared with 28.9% of participants given a placebo gel. 

Among secondary goals, the time to first wound closure was a mean of 37.7 days in the Filsuvez group and 44.5 days in the control group. However, the proportion of wholly closed wounds and wound healing over three months were not significantly different.

Participants reported less pain during dressing changes with Filsuvez, which was statistically significant on day 14 and showed a trend toward benefits at three months.

Filsuvez was safe and well-tolerated, with the most frequently reported side effects being wound complications, fever, wound infection, itching, and anemia (low levels of healthy red blood cells).

Earlier this year, the FDA granted orphan drug designation to the company’s AP103, a potential topical gene therapy for dystrophic EB.