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Santhera facing long delay for muscular dystrophy drug in US

FDA rejects accelerated marketing application for Raxone to await phase II trial results

SantheraSanthera is facing a considerable delay in the US before it can get approval for Duchenne muscular dystrophy (DMD) candidate Raxone.

The Swiss biopharma company has failed to convince the FDA to accept an accelerated marketing application for Raxone (idebenone) via the truncated subpart H approval pathway, used to hasten the approval of drugs for serious and rare diseases with inadequate treatment options.

The decision is the latest in a string of disappointments for DMD patients, who have now seen four drugs come in front of the US regulator only to be delayed or rejected and still have no approved therapy for their illness.

Santhera has hoped to use the truncated approval pathway to gain approval for Raxone in DMD patients not taking glucocorticoid drugs based on one already-completed trial, whilst carrying out a second confirmatory study.

The US regulator does not agree however, and says it wants to wait for the results of the SIDEROS trial, which will test the drug in DMD patients regardless of whether they are taking glucocorticoids.

That trial has not yet started and is not due to complete until 2019 so could delay approval of Raxone in the US until 2020. A marketing application in the EU was however accepted by the EMA last month, which could lead to a possible approval early next year.

"We are disappointed that the FDA does not support our plan to file … for Raxone," said Santhera chief executive Thomas Meier. However, he added the company will take comfort from the "clarity" of the FDA that SIDEROS will allow it to file for approval in all DMD patients, regardless of glucocorticoid use.

Shares in the company saw a third wiped off their value ahead of the weekend as news of the setback emerged, but DMD patients in the US must be despairing of getting access to new therapies for the disease.

In recent months the FDA has rejected BioMarin's exon-skipping therapy Kyndrisa (drisapersen) - which has now been abandoned after also being turned down by the EMA - as well as PTC Therapeutics' Translarna (ataluren), which is already on the market in Europe and has been backed by the UK's National Institute for Health and Care Excellence (NICE).

The US agency has also asked for new data on Sarepta's exon-skipping drug eteplirsen that has extended the already-delayed review beyond the new deadline of 26 May, although on a more encouraging note the drug is at least still in the running for an approval in the near-term.

Unlike these three drugs Raxone does not seek to modify the underlying disease mechanism in DMD, but according to clinical trial results helps improve lung function in DMD patients, tackling one of the key complications of the muscle-wasting disease.

Article by
Phil Taylor

18th July 2016

From: Regulatory

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