MHRA extends Santhera’s Puldysa on the Early Access to Medicines Scheme

pharmafile | June 23, 2020 | News story | Manufacturing and Production  

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has extended Santhera’s Duchenne muscular dystrophy (DMD) drug Puldysa (idebone) for a further year on the Early Access to Medicines Scheme (EAMS).

This will help DMD patients with respiratory function decline to continue to access the drug. The condition is a leading cause of increased morbidity and early death, with no approved therapy currently existing.

This move shows the MHRA have a positive scientific opinion for the drug that is currently having its marketing authorization application reviewed by the European Medicines Agency. A conditional marketing authoriatisation for the drug is being reviewed by the Committee for Medicinal Products for Human Use (CHMP) with an expected decision coming in the fourth quarter of 2020.

Kristina Sjöblom Nygren, Santhera’s Chief Medical Officer and head of development, said: “This EAMS renewal for idebenone enables a much-needed therapeutic option for DMD patients with deteriorating respiratory function who have no real treatment alternative. We welcome the continued recognition by the UK’s MHRA of the positive benefit-risk of idebenone in this patient population.”

DMD patients have been able to access the treatment through EAMS since June 2017. 84 patients with the condition currently benefit from the treatment in specialised DMD centres across the UK.

Conor Kavanagh

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