Chiesi Global Rare Diseases won approval from the U.S. Food and Drug Administration (FDA) on Dec. 19, 2023, for Filsuvez^® (birch triterpenes) Gel. It treats patients as young as six months old for partial thickness skin wounds associated with two subtypes of epidermolysis bullosa (EB) – junctional (JEB) and dystrophic (DEB). Rare inherited conditions, cause light or moderate touching to produce widespread blisters that affect the skin and mucous membranes – often resulting in open sores that can be painful and easily infected. Patients who have severe EB, especially JEB, may not survive beyond their first two years of life. Filsuvez will be applied to open sores and then covered with a soft, nonadhesive dressing. It should be used every time that the covering is changed until the sore heals. Plans for its launch, distribution, and pricing are not yet available. Full prescribing information is here.

At a Glance

1. Brand (Generic) Name: Filsuvez (birch triterpenes) Gel
2. Manufacturer: Chiesi Global Rare Diseases
3. Date Approved: Dec. 19, 2023
4. Indication: for the treatment of wounds associated with DEB and JEB in patients aged six months and older
5. Dosage Forms Available: single-dose sterile tubes containing 25mL of 10% Filsuvez gel
6. Launch Date: Not available

• Estimated Annual Cost: Not available
• EB includes a group of four genetic conditions that weaken skin and mucous membranes, sometimes to the point that even a soft touch can cause extensive blistering. Patients who have severe forms of the condition may also have anemia, lack of normal growth, malnutrition, scarring, and missing fingernails and toenails. They are more likely to develop skin cancers.
• Approximately one infant in every 20,000 who are alive at birth has some form of EB – about 200 babies per year in the U.S. Not all cases are life-threatening, but children who have severe EB – particularly JEB – may die before the age of two years old.
• Of 223 participants in the EASE clinical trial, 41% of those using Filsuvez saw complete healing of treated sores within 45 days as opposed to 28.9% of patients using an identical gel base without any active drug. Dressings were changed between once a day and once every four days.
• The main side effects reported during the study were itching and pain at the places where either Filsuvez or the placebo gel was applied.
• Current treatment for EB centers on preventing blister formation by avoiding heat, minimizing skin contact, and wearing soft loose-fitting clothing.
• Some patients need supplemental treatments for anemia, electrolyte imbalances, and vitamin or mineral deficiencies.
• Krystal Biotech’s Vyjuvek^™ (beremagene geperpavec-svdt) topical gel was approved by the FDA on May 19, 2023. The topical, revocable gene therapy is approved for the treatment of wounds for patients six months of age and older who have DEB with specific mutations. A healthcare professional, either at a healthcare clinic or in a home setting, applies Vyjuvek once per week.