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CHMP hands Santhera second ‘no’ in Duchenne muscular dystrophy

Swiss biopharma says it will re-file Raxone for the licence extension

SantheraEuropean regulatory advisers have refused to shift their negative position on Santhera’s Duchenne muscular dystrophy (DMD) drug Raxone (idebenone).

The Committee for Medicinal Products for Human Use (CHMP) rejected the recommendation of the orphan drug, which was indicated for DMD patients to slow their gradual loss of breathing ability.

It’s the second such knock-back for Raxone’s licence extension and shares in the Swiss firm plummeting by almost a third last week after it warned of a likely negative CHMP verdict.

Despite the Committee deciding that approval could not be granted based on current existing evidence, it did acknowledge the positive outcome of the phase III DELOS trial, which demonstrated statistically significant evidence that Raxone slows the decline of respiratory function amid other improvements.

In response, Santhera said it would continue working with regulatory authorities to “bring this treatment option to patients as quickly as possible”.

Thomas Meier, chief executive officer of Santhera, said: “Data from the phase III DELOS trial demonstrated statistically significant and clinically relevant evidence that Raxone slows the decline of respiratory function, and reduces the risk of bronchopulmonary complications and hospitalisation in patients with DMD not using glucocorticoids.”

Respiratory decline is one of the leading causes of death in DMD patients and, according to Thomas Voit, professor of paediatrics at London’s Great Ormond Street Hospital, about half of patients with DMD aged 10-20 years old aren’t able to take steroids or have had to cease use due to side effects.

Commenting on the decision, Nic Bungay, director of campaigns for Muscular Dystrophy UK, said: “This is disappointing news for the people with DMD who could benefit from Raxone and with no alternative treatment to help preserve respiratory function when steroids stop working, patients can struggle to cough and clear their airways.”

Until now, Raxone was made available to patients via the early access medicines  scheme (EAMS) under an ‘exceptional circumstance’, which comes about when an applicant can show they are unable to provide efficacy and safety data for the candidate but it is approved anyway due to the rarity of the condition it is intended for.

There has been no word on whether patients currently taking the drug can continue via EAMS however, but Muscular Dystophy UK has suggested they will urge the Medicines and Healthcare products regulatory agency (MHRA) to ensure patients can continue with the medication.

Article by
Gemma Jones

29th January 2018

From: Regulatory



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