Experimental ALS Drug OK’d For Some Worst-Case Patients
Biogen's tofersen drug will be allowed for some patients dying of ALS after an important study concludes this summer. Also in pharmaceutical news: Pharma's reputation, microbiome therapeutics and a patent battle in Australia.
Bloomberg:
Biogen To Make Experimental ALS Drug Available To Dying Patients
Biogen Inc. said it will make an experimental medicine for Lou Gehrig’s disease available to some people who are dying of the incurable illness starting in July, following months of pressure from patients who had no other treatment options. The drug, known as tofersen, will be offered to the most rapidly progressing patients after researchers complete a key study this summer, the company said in a statement posted on its website. Tofersen hasn’t been reviewed or approved by regulators in any country. It will be given on a compassionate-use basis after everyone who was given a placebo during the clinical trial has been offered the medicine, Biogen said. (Fay Cortez and LaVito, 4/26)
In other pharmaceutical industry news —
Stat:
Pharma's Reputation Among Patient Groups Is Climbing
Thanks to the rapid development of Covid-19 vaccines, nearly two-thirds of patient advocacy groups believe the pharmaceutical industry was effective at tackling the pandemic, boosting its reputation last year to the highest level in a decade. But at the same time, a majority of the groups also found that, other than research and development, drug makers were lacking in most other areas of operation. (Silverman, 4/26)
Stat:
Microbiome Therapeutics Drugs Still Face A Wave Of Challenges
Even as drug makers are poised to introduce actual medicines to change a person’s microbiome and make them healthier, there’s a lot we don’t know about the billions of organisms that live inside us. We don’t know all their names, let alone everything they’re capable of doing. In a deep new report, STAT examines what we do know about this field — the science powering this new array of therapies, the companies already charging toward new drug applications, and, critically, the many challenges still to come. (Sheridan, 4/26)
Modern Healthcare:
Employers, Payers Seek Transparent Pharmacy Benefit Models
More employers and healthcare payers are carving out their pharmacy benefit management as they seek more transparency, business groups said. Health plans typically administer pharmacy benefit services internally or contract pharmacy benefit managers, which negotiate rebates and discounts with drug manufacturers and pocket an undisclosed share. More employers and payers are contracting directly with PBMs, increasingly working with more transparent managers that pass all the drug rebates and discounts to employers and payers for a set fee. (Kacik, 4/26)
Stat:
Bristol Myers Squibb Fights An 'Unreasonable' Australian Law Over Patents
Two big drug makers are challenging a provision in Australian patent law that they argue is “unreasonable,” and the outcome could determine the extent to which the pharmaceutical industry continues to find the Australian market attractive. In a recent complaint, Bristol Myers Squibb (BMY) and Ono Pharmaceutical (OPHLY) maintain that existing law unfairly restricts the ability of a company to win a five-year patent extension for a product and, moreover, is out of step with the prevailing standards in many other countries. (Silverman, 4/26)
