EMA Committee Recommends New Therapies Including Vyjuvek for Rare Skin Disease and Expands Existing Drug Indications

Key Insights

• The EMA's Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion for Vyjuvek (beremagene geperpavec) to treat wounds in patients with dystrophic epidermolysis bullosa of all ages.
• The Committee confirmed its previous marketing authorization recommendation for Leqembi (lecanemab) remains valid, while also expanding the indication for the chikungunya vaccine Ixchiq to include adolescents from 12 years.
• Fabhalta received approval for treating adult patients with complement 3 glomerulopathy, and Deqsiga was recommended for approval in primary and secondary immunodeficiencies.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has announced several significant regulatory recommendations, expanding treatment options across multiple therapeutic areas.

Breakthrough for Rare Genetic Skin Condition

In a notable development, Vyjuvek (beremagene geperpavec) received a positive opinion for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB), a rare and devastating genetic skin condition. This recommendation spans all age groups, potentially offering hope to patients who currently have limited treatment options.

Alzheimer's Treatment Status Confirmed

The CHMP has reaffirmed its position on Leqembi (lecanemab), stating that its previous opinion recommending marketing authorization for the Alzheimer's treatment remains unchanged and does not require updates. This confirmation provides clarity for healthcare providers and patients regarding the medication's regulatory status in Europe.

Expanded Vaccine Coverage for Chikungunya

In a move to enhance protection against infectious diseases, the committee approved an extension to the existing indication for the chikungunya vaccine Ixchiq. The vaccine's use will now include active immunization of adolescents from 12 years of age, broadening the protected population against this mosquito-borne viral disease.

New Therapeutic Options in Rare Diseases and Immunology

The committee delivered positive opinions for two additional treatments. Fabhalta received approval for an extended indication to treat adult patients with complement 3 glomerulopathy, a rare kidney disease. Additionally, Deqsiga garnered a recommendation for approval in treating both primary and secondary immunodeficiencies, as well as for immunomodulation in autoimmune diseases.

These recommendations reflect the EMA's ongoing commitment to advancing therapeutic options across various medical conditions, from rare diseases to more common immunological disorders. Once implemented, these decisions will significantly impact treatment paradigms and patient care across Europe.