RARE Daily

UK MHRA Approves Nulibry to Treat Adult Patients with MoCD Type A

April 10, 2024

Rare Daily Staff

The UK Medicines and Healthcare products Regulatory Agency approved BridgeBio subsidiary Origin Biosciences’ Nulibry to treat adult patients in Great Britain with the genetic disease molybdenum cofactor deficiency Type A.

Molybdenum cofactor deficiency (MoCD) Type A is a rare genetic disorder in which the body is not able to produce cyclic pyranopterin monophosphate (cPMP). This causes a build‐up of a sulphite which is toxic and damages the brain. MoCD is usually diagnosed in babies once they are born and causes symptoms such as seizures, involuntary movements and difficulties in feeding.

The MHRA’s approval relied on a European Commission authorization in September 2022 that was supported by data from three clinical trials that demonstrated the efficacy of Nulibry for the treatment of patients with MoCD Type A compared to data from a natural history study.

“Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us,” said Julian Beach, MHRA interim executive director, Healthcare Quality and Access. “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.”

MoCD type A results from an error in the gene called MOCS1. This stops the body from producing cPMP. When this substance is missing, certain compounds (sulphites) formed in the body cannot be broken down. These compounds are toxic to the brain and can negatively affect or delay the development of a child. Nulibry (fosdenopterin) provides the missing substance that the patient’s body needs to break down the harmful sulphite compounds.

The benefits of fosdenopterin were seen in five main studies involving a total of 52 patients with MoCD type A. The studies looked at the effect of fosdenopterin on survival after one year of treatment. The results in the 15 patients treated with fosdenopterin were compared with historical data from two studies involving 37 patients who did not receive fosdenopterin or any other treatment.

After one year, around 93 percent of patients using fosdenopterin were alive compared with around 75 percent of those who received no treatment. The studies also indicated that early 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.  The most common side effects of the medicine are related to the injection device, and include pain, discharge, redness, and inflammation.

The new marketing authorization was granted to TMC Pharma. Nulibry, originally developed by Alexion, was sold to BridgeBio affiliate Origin Biosciences in 2017. It became BridgeBio’s first therapeutic to be approved in the United States in February 2021. Global rights were sold to Indian biotech Sentynl in early 2022 and it was approved for use in the European Union in September of that year.

Photo: Julian Beach, MHRA interim executive director, Healthcare Quality and Access

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