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Biogen will offer expanded access in mid-July, which may be too late for Lisa Stockman Mauriello.
Biogen will launch an expanded access program for its investigational amyotrophic lateral sclerosis (ALS) drug tofersen in July — but that’s likely too late for the patient whose quest to try the therapy has brought it into the spotlight.
In a “community update” posted to its website, Biogen said it will offer compassionate use to the “most rapidly progressing patients” starting in mid-July, once placebo patients in its phase III trial are transitioned to active therapy.
The company noted that this would be “before the drug has been confirmed to be safe and effective.” It defined “rapidly progressing” as SOD1-ALS patients with an ALS Functional Rating Scale-Revised (ALSFRS-R) slope decline greater than or equal to 2 points per month, and said access would be confirmed by a third-party organization “in an anonymized manner.”
Then, in the fall, if study data are positive and no additional trials are required, the company said it will launch a full expanded access program for all SOD1-ALS patients.
isa Stockman Mauriello, the SOD1-ALS patient whose grassroots campaign raised awareness about access to tofersen, issued a statement Tuesday morning, highlighting her disappointment that access would likely come too late for her.
Stockman Mauriello is among the 2% of ALS patients with a SOD1 mutation, of which there are many types. She has the one known as A5V, which is one of the most rapidly progressive forms among these already rapid progressors. Enrollment in the phase III trial had just closed when she was diagnosed in January.