The AMDM announcement dated July 24 notes that the list of registered drugs includes some new ones. These are intended for the treatment of rare diseases. For example, Miglustat Dipharma (international nonproprietary name — miglustat), is indicated for the treatment of Type I Gaucher disease in adults when enzyme replacement therapy is not possible, as well as for the neurological manifestations of Type C Niemann-Pick disease in adults and children.
The drug Nitisinone Dipharma (INN—nitisinone) has also been registered; it is used to treat type I hereditary tyrosinemia in patients of all ages and alkaptonuria in adults. Treatment is administered in combination with a special diet prescribed by a physician.
The Agency noted that the decisions to register these drugs were made unanimously by the members of the Commission.
