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Biogen to open compassionate use programme for ALS patients with ‘rapidly progressive disease’

Move comes after mounting pressure from patients and campaigners

Biogen has announced that it will offer its investigational drug tofersen to patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) who have no other options from mid-July.

The move follows pressure from ALS campaigners and patients to open access to the medicine, which is still being evaluated in a phase 3 clinical trial.

In a statement, Biogen said that although it does not "believe it is fair to ask participants in this [phase 3] study to continue to receive placebo while other SOD1-ALS patients are offered access to tofersen […], access could be provided as soon as the placebo-controlled study has ended".

"Until the safety and efficacy have been established, we will prioritise early access for a subset of the most rapidly progressing patients with this rare, severe disease," the company added.

Biogen is aiming to start offering compassionate use access to a subset of SOD1-ALS patients with the ‘most rapidly progressive disease’ after patients in the late-stage study on placebo transition to tofersen treatment.

This stage will come before the safety and efficacy of the drug are established, Biogen noted.

Following this, if the results from the phase 3 study show that tofersen is safe and effective and no additional studies are needed, Biogen will then launch an early access programme (EAP) for the broad SOD1-ALS population from autumn 2021.

The EAP for tofersen will start before Biogen submits applications for regulatory approval of the drug.

Previously, Biogen had refused to provide expanded access to tofersen, mainly on the grounds that this would "risk failing to complete the study and risk failing to obtain access for all SOD1-ALS patients".

In response, the patient movement I AM ALS commented: “We vehemently refute the notion that an expanded access programme cannot be conducted in parallel with a clinical trial, and reject the notion that doing so poses an ethical issue.”

“In fact, keeping a therapy that can only reach someone who doesn’t qualify for a trial through the provision of expanded access from them poses a far greater ethical concern.”

With the pressure mounting, Biogen has finally expanded access to the drug but is urging caution, saying “while we hope this study’s results will be positive, we [Biogen] know firsthand that promising drugs can fail in phase 3 studies”.

Article by
Lucy Parsons

27th April 2021

From: Healthcare

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