The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated with Niemann-Pick disease type C in patients aged 2 years and older.
Miplyffa (arimoclomol; Zevra Therapeutics), the first drug approved to treat the condition, is an oral therapy that can be administered three times a day with or without food, by either the patient or caregiver, according to a press release from the manufacturer. Precise dosing, ranging from 47 mg to 124 mg, will be dependent upon the patient’s body weight, with follow up by a health care provider, the company