EMA Seeks Expert Input Before Deciding on Filsuvez Gel for Wounds

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by Marta Figueiredo PhD |

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The European Medicines Agency (EMA) is seeking the opinion of independent epidermolysis bullosa (EB) experts and patients before issuing its recommendation on the potential approval of Amryt Pharma’s topical gel Filsuvez (Oleogel-S10) to treat skin wounds in people with junctional EB (JEB) and dystrophic EB (DEB).

Given EB’s rarity and lack of approved therapies, this specific advice is expected to help the agency in its decision.

As such, a recommendation by the EMA’s Committee for Medicinal Products for Human Use (CMHP) is now expected in March; it previously had been anticipated by January. CHMP opinions are generally accepted by the European Commission, which makes final decisions on therapy approval for the 27-state union.

“We welcome the input from independent EB physicians and importantly the voice of EB patients, via the Ad-Hoc Expert Group to assist the EMA progress its regulatory assessment of [Filsuvez],” Joe Wiley, Amryt’s co-founder and CEO, said in a press release.

“If approved, we are confident in the potential for [Filsuvez] to be an effective therapy for patients suffering from this terrible condition and are ready to launch what will be the first to market novel therapy in EB,” Wiley added.

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Notably, this marks the second delay in Filsuvez’s review process, initiated in March 2021. The first, pushing a recommendation from late 2021 to January, was associated with the need for Amryt to address a list of outstanding questions issued by the EMA that included major objections to the therapy’s approval.

This latest two-month delay comes on the heels of the U.S. Food and Drug Administration (FDA) also pushing back by three months its decision on Filsuvez’s approval for the same indication in the United States. The FDA wanted more time to review additional data submitted by Amryt in support of its application.

That application to the FDA was granted priority review in June, and a final decision is now expected no later than Feb. 28 under its set action date.

Filsuvez received orphan drug status in both the U.S. and Europe, and fast track and rare pediatric disease designations in the U.S. These designations are meant to help accelerate the therapy’s clinical development and regulatory review.

Originally developed by Birken AG, which was later acquired by Amryt, Filsuvez is a gel applied to the skin that contains birch bark extract, which is rich in a compound called betulin. Evidence suggests that betulin can activate skin cells and regulate the inflammatory response involved in wound healing.

In Europe, the gel was approved in 2016 under the brand name Episalvan for the treatment of partial-thickness skin wounds — those in which the top layers of the skin have been lost due to burns or skin grafts.

The current regulatory applications are mainly based on positive top-line data from the global Phase 3 EASE trial (NCT03068780), which evaluated Filsuvez’s safety and effectiveness against a placebo gel in 223 EB patients. That study was the largest in EB, according to Amryt.

Specifically, the trial enrolled 156 children and 67 adults with JEB, DEB, or Kindler syndrome — a very rare form of the disease — who had an EB-related wound between 10 and 50 square centimeters in size (about four by 20 square inches) for at least three weeks, but less than nine months.

Participants were randomly assigned to treat their wounds with either Filsuvez or a placebo gel for three months.

Top-line results showed that the trial met its main goal, with a significantly greater proportion of Filsuvez-treated patients showing wound closure within 45 days than those receiving a placebo gel (41.3% vs. 28.9%).

In contrast, differences in secondary goals — including the proportion of participants with complete wound closure after 90 days — did not favor Filsuvez significantly, although favorable trends were observed.

The therapy was generally safe and well-tolerated, with the most frequently reported adverse events being wound complications, fever, wound infection, itching, and low levels of red blood cells.

All patients who completed the three-month period entered the study’s extension phase, in which all are receiving Filsuvez for two years to collect long-term safety data.