The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a marketing authorization for Yargesa (miglustat), for the treatment of adults with mild-to-moderate type 1 Gaucher disease.
Yargesa is a generic of Zavesca, which has been authorized in the European Union since November 2002. Yargesa has demonstrated satisfactory bioequivalence to Zavesca, which is why it is now applying for marketing authorization.
The new medicine will be available as capsules of 100 mg of its active substance, miglustat. This active substance is an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of glucocerebroside.
Yargesa works as substrate reduction therapy, meaning that it reduces production of glucocerebroside, one of the substrates of the defective enzyme (glucocerebrosidase) in patients with Gaucher disease. This reduction is expected to slow down, or prevent, symptoms of type 1 Gaucher disease.
Full indication for Yargesa states that the medicine is meant for the oral treatment of adult patients with mild-to-moderate type 1 Gaucher disease, and that it may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.
Detailed recommendations for the use of Yargesa will be described in the summary of product characteristics (SmPC), which will be published soon by the European Commission.
According to a press release, at the Jan. 26 meeting where the European Medicines Agency’s (EMA) CHMP decided to recommend Yargesa, seven other medicines also were recommended for approval, including two biosimilars.
Xeljanz (tofacitinib) was recommended for a marketing authorization for the treatment of rheumatoid arthritis; Jylamvo (methotrexate) received a positive opinion for the treatment of rheumatological disorders and psoriasis and for the maintenance of acute lymphoblastic leukaemia (ALL); Rolufta (umeclidinium) was recommended for the treatment of chronic obstructive pulmonary disease (COPD); and Tadalafil Lilly (tadalafil) was recommended for the treatment of erectile dysfunction and signs of benign prostatic hyperplasia.
Two biosimilars were recommended: Amgevita and Solymbic, both containing the active substance adalimumab, were recommended for several indications, including inflammatory bowel diseases (IBDs) and some forms of arthritis.
Besides Yargesa, another generic medicine, Daptomycin Hospira (daptomycin) was recommended for the treatment of complicated skin and soft-tissue infections (cSSTI), right-sided infective endocarditis (RIE) due to staphylococcus aureus and S. aureus bacteraemia associated with RIE or with cSSTI.