Is There a Right or Wrong in the Debate Between Patients and Manufacturers to Release Drugs Through Compassionate Use?


Following several Patient Worthy articles on the subject earlier this year, Fierce Biotech recently published news that Biogen is preparing to release the Phase III VALOR trial drug, tofersen, in mid-July prior to determining the trial’s final results. Tofersen is being developed to treat amyotrophic lateral sclerosis (ALS).

Biogen’s discussions with ethicists, patient advocates, regulators, and trial investigators contributed to its decision for early release. Biogen sent a letter to that effect to the ALS community.

At the end of its letter, Biogen issued a “reminder” that even drugs with potential can fail in the final Phase III study. The failure of its drug dexpramipexole led to its focus on drugs like tofersen that featured anti-inflammatories.

However, as a result of her rapidly deteriorating condition, Lisa Stockman Mauriello, an ALS SOD1 A5V patient, was at the forefront of the effort to release the drug. The A5V mutation is deadly and overall survival has generally been twelve months.

Although she is pleased with Biogen’s decision, Lisa is concerned that it may be too late for patients like herself.

Ironically, only one day before sending out its new early-release plan, Biogen had denied Lisa’s request for expanded access (compassionate use) for the fifth time.

About The Tofersen Expanded Access (Compassionate Use) Program

Doctors may begin to apply for the drug on behalf of their eligible patients effective June 25th. The drug will be ready to ship to approved locations around mid-July.

Tofersen is an antisense oligonucleotide (ASO) against the superoxide dismutase 1 (SOD1) gene. Its mechanism reduces the production of the SOD1 protein slowing the progression of the disorder.

Biogen’s new program for the distribution of tofersen is two-fold. The first segment will involve switching trial patients from the placebo to tofersen. At this point, the safety and efficacy of the drug will still not have been established. Nevertheless, the drug will be distributed to the patients with the most severe disease progression.

Upon proof of tofersen’s safety and effectiveness, Biogen will allow access to the broader SOD1 ALS community this fall under an Early Use Program.

Lisa’s Proposal

In Lisa’s fifth request, she suggested that for reasons of ethical concern, patients with her specific mutation could be randomized to receive tofersen or a placebo in a pattern similar to that of the Phase III VALOR trial participants. Then new participants would have the same 67% odds of receiving tofersen. Her letter goes on to say that in mid-July via early access, all patients receiving the placebo would then be switched over to the drug at the same time.

Looking Forward

Biogen now has four ASOs for ALS in its pipeline. Therefore, there is a tremendous amount of interest in the results of the VALOR trial which should be completed by the end of August 2021.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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