Miglustat

IntraBio’s green light likely comes as a blow to Zevra Therapeutics, which snagged a historic approval for Miplyffa in Niemann-Pick disease type C on Friday. While it was just last week that Zevra Therapeutics’ Miplyffa became the first FDA-approved treatment for the lysosomal storage disorder Niemann-Pick disease type C (NPC), IntraBio has already one-upped its rare disease rival.Late Tuesday, the FDA approved IntraBio’s levacetylleucine under the brand name Aqneursa to treat neurological symptoms of NPC in adults and kids weighing at least 15 kg (about 33 pounds).Aqneursa has been approved as a standalone therapy and is available now, according to a release from IntraBio—which hails from the U.K. but recently shifted its headquarters to Austin, Texas, following a $40 million funding round.The green light likely comes as a blow to Zevra, which snagged a historic approval for Miplyffa in NPC on Friday. But, unlike IntraBio’s monotherapy, Miplyffa must be taken with the enzyme inhibitor miglustat. What’s more, Miplyffa isn’t expected to hit the market for another eight to 12 weeks, according to Zevra.In turn, IntraBio’s approval and launch plan have effectively erased Zevra’s head start. NPC, a rare lysosomal storage disorder, prevents the body from moving cholesterol and other lipids in cells. This leads to a buildup of cholesterol and other fats in the liver, spleen or lungs that can cause organ damage. On average, people with NPC only live for about 13 years, the FDA noted in its release.The disease can also impair patients’ speech, cognition, swallowing, movement and fine motor skills.The FDA based its approval of Aqneursa on results from IntraBio’s pivotal trial IB1001-301, which looked at the drug’s effect on neurological symptoms and functioning in adults and kids over the age of 4 with a confirmed NPC diagnosis.The trial met its primary efficacy endpoint and every secondary endpoint across all patient cohorts receiving Aqneursa, IntraBio said. The study found that Aqneursa “significantly” improved neurological signs and symptoms of the disease and led to functional benefits within 12 weeks.The main outcome the FDA looked at in the study was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA)—known as the functional SARA (fSARA)—which measures gait, stability, speech and upper and lower limb coordination across eight individual domains. The fSARA, meanwhile, only assesses gait, sitting, stance and speech disturbance domains with modifications to the scoring responses.Patients on Aqneursa showed greater improvement on fSARA scores versus those on placebo, and the results on the modified assessment were buoyed by consistent outcomes demonstrated in the original test, IntraBio explained.While the most common side effects associated with Aqneursa are abdominal pain, difficult swallowing, upper respiratory tract infection and vomiting, the drug’s prescribing information does carry a warning that Aqneursa may cause embryo-fetal harm if used during pregnancy.Patients should inform their doctors of a known or suspected pregnancy before taking IntraBio’s drug, the FDA noted.Aqneursa is taken orally up to three times a day, with or without food. The dosing varies depending upon the individual patient’s body weight.Aqneursa marks IntraBio’s first commercial product and, with an approval in hand, the company isn’t content to stop at NPC.“Based on our clinical research, we believe that Aqneursa may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop Aqneursa for these additional indications,” Mallory Factor, CEO of IntraBio, said in a statement. In order to help patients access the treatment, IntraBio has launched a support program dubbed Aqneursa Cares. The program will include financial support to “reduce or eliminate” out-of-pocket costs for qualifying patients as well as connect them with third-party resources. The program will also feature educational resources and a team of disease specialists.

Within the span of a week, the FDA gave the green light to another drug for the rare condition Niemann-Pick disease type C in the form of IntraBio’s levacetylleucine. On Friday, the FDA approved Zevra Therapeutics’ Miplyffa for NPC when added to Johnson & Johnson’s enzyme inhibitor Zavesca. Zevra had resubmitted the thrice-daily pill with a reanalysis of its registrational trial after its initial attempt at approval was rejected in 2021. The approval of IntraBio’s levacetylleucine, which will be marketed as Aqneursa, was based on data from a 60-participant Phase 3 trial that showed the treatment achieved a “clinically meaningful” 1.37-point reduction on the Scale for the Assessment and Rating of Ataxia (SARA) score at 12 weeks versus placebo (p<0.001). SARA measures a patient’s level of muscle control when doing activities like walking, sitting and standing in place. Aqneursa, which is a modified amino acid drug, is indicated for both adults and children weighing at least 15 kg. The oral treatment can be taken up to three times per day with dosing dependent on patient weight. The back-to-back authorizations reflect the FDA’s “commitment to support development of new treatments for rare diseases,” Janet Maynard, director of CDER’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in a Tuesday release. Prior to these approvals, the only options to manage NPC were pain medications, physical therapy and speech therapy, among other strategies. Zavesca, which is approved for certain patients with Gaucher disease in the US, is also an off-label option. Driven by changes in the NPC1 or NPC2 genes, NPC causes poor muscle tone, difficulty coordinating movements and progressive organ damage. There are around 300 to 350 people with the condition in the US, according to William Blair analysts.