EU Approves Amicus Therapeutics Pombiliti for Late-Onset Pompe Disease
The European Commission has approved Amicus Therapeutics’ Pombiliti (cipaglucosidase alfa) as a long-term enzyme replacement therapy for adult patients with late-onset Pompe disease, a rare neuromuscular disorder that affects skeletal muscle and breathing.
The commission’s decision follows a positive opinion of Pombiliti in December 2022 from the EMA’s Committee for Medicinal Products for Human Use (CHMP), based on positive results from a late-stage study.
Pombiliti must be administered along with miglustat, an enzyme inhibitor. The CHMP is expected to issue a separate opinion on miglustat in the second quarter of this year.
On 15 December 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Pombiliti, intended for the treatment of glycogen storage disease type II (Pompe disease). The applicant for this medicinal product is Amicus Therapeutics Europe Limited.
About Pombiliti
On 15 December 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Pombiliti, intended for the treatment of glycogen storage disease type II (Pompe disease). The applicant for this medicinal product is Amicus Therapeutics Europe Limited.
Pombiliti will be available as a 105 mg powder for concentrate for solution for infusion. The active substance of Pombiliti is cipaglucosidase alfa, a recombinant human acid α-glucosidase (ATC code: A16AB23), which is an enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.
The benefit of Pombiliti is its ability to improve the motor function (six‑minute walk distance) of patients with late-onset Pompe disease when used in combination with miglustat. The most common side effects are infusion-associated reactions such as chills, dizziness, urticaria, flushing, somnolence, anaphylaxis, chest discomfort, cough, infusion site swelling, and pain.
Amicus Therapeutics is a public American biopharmaceutical company based in Philadelphia, PA. The company went public in 2007 under the NASDAQ trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX. Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates.
March 30, 2023
