US FDA Approves IntraBio’s Drug for Rare Genetic Disease

(Reuters) -The U.S. Food and Drug Administration approved IntraBio’s drug for a rare and fatal genetic disorder, the health regulator said on Tuesday, just days after the agency cleared the first treatment for the disease.

Niemann-Pick disease type C (NPC) is a rare genetic disorder that affects the nervous system and other organs, causing physical and mental disabilities such as speech issues, difficulties with swallowing and coordination, clumsiness and others over time.

NPC can occur between infancy and adulthood, which is why it is sometimes called “childhood Alzheimer’s disease.” People affected by it only live for about 13 years.

The company estimates that NPC affects one in 100,000 live births.

On Sept. 20, the FDA approved Zevra’s drug, Miplyffa, for the treatment of NPC – the first treatment to get a nod for the condition.

The FDA approved the drug for the treatment of neurological symptoms associated with NPC in adults and pediatric patients weighing at least 15 kilograms.

IntraBio’s oral drug, called IB1001, will be sold under the brand name Aqneursa. However, the regulator did not disclose the price of the drug.

On Monday, Zevra said it would price its rival treatment, Miplyffa, at a wholesale acquisition cost of between $40,000 and $106,000 per month, depending on dosage.

Aqneursa should be taken orally up to three times per day, with or without food. The recommended dose varies depending on the individual’s body weight.

The approval was based on a late-stage clinical trial in 60 patients that showed the drug helped reduce symptoms compared to a placebo.

The FDA also put out a warning that Aqneursa may cause embryo-fetal harm if used during pregnancy.

The Austin-headquartered company said in March it had raised over $40 million in equity funding to support the commercialization and launch of IB1001, subject to FDA approval.

(Reporting by Sruthi Narasimha Chari in Bengaluru; Editing by Alan Barona)