With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.

Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for Niemann-Pick disease type C (NPC), a rare genetic disease linked to progressive neurological symptoms that is almost always fatal. Branded Miplyffa, it is indicated for use in combination with enzyme inhibitor miglustat in adults and children, ages 2 and older.

Zevra Therapeutics Inc. will acquire Acer Therapeutics Inc. in a $91 million deal that combines two companies with expertise in rare diseases. Acer has struggled since its hot flash drug, ACER-801 (osanetant), failed to hit statistical significance in a phase IIa proof-of-concept study in March 2023. The stumble came amidst the launch of Acer’s Olpruva, a sodium phenylbutyrate for oral suspension therapy approved by the U.S. FDA in late December 2022 for treating urea cycle disorders. The day before the Zevra merger was announced, Acer reacquired the worldwide development, commercialization and economic rights to Olpruva from its development partner, Relief Therapeutics Inc.