The Medicines and Healthcare products Regulatory Agency (MHRA) has approved TMC Pharma’s Nulibry (fosdenopterin) to treat patients with the rare genetic disease molybdenum cofactor deficiency (MoCD) type A.
Estimated to affect one in 100,000 to 200,000 newborns worldwide, MoCD occurs when the body is not able to produce cyclic pyranopterin monophosphate. This causes a build‐up of a sulphite, which is toxic and damages the brain, resulting in symptoms such as seizures, involuntary movements and difficulties feeding.
Fosdenopterin, administered intravenously, provides the missing substance that MoCD patients need to break down the harmful sulphite compounds.
The MHRA’s approval of the therapy occurred through the European Commission Decision Reliance Procedure and was supported by positive results from five studies involving 52 patients with MoCD type A.
The results of the 15 fosdenopterin-treated patients were compared with historical data from two studies involving 37 patients who did not receive fosdenopterin or any other treatment.
After one year, approximately 93% of patients receiving fosdenopterin were alive, compared with about 75% of those in the non-treatment group. The studies also demonstrated that beginning treatment with fosdenopterin before patients develop major brain damage preserves the ability to take food by mouth and improves growth and development of motor and cognitive functions.
Julian Beach, MHRA interim executive director, healthcare quality and access, said: “Keeping patients safe and enabling their access to high-quality, safe and effective medical products are key priorities for us.
“We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all products, we will keep its safety under close review.”
The authorisation comes just days after the MHRA approved Advanz Pharma’s combined antibiotic, Exblifep (cefepime/enmetazobactam), to treat adults with urinary tract infections (UTIs), certain pneumonia types acquired during hospital stays and bacteraemia.
This approval occurred through the regulator’s new International Recognition Procedure and was supported by evidence from a study involving 1,041 adults that showed that Exblifep was more effective than Pfizer’s combined antibiotic, Tazocin (piperacillin/tazobactam), when treating complicated UTIs, including acute pyelonephritis.