Despite the approval of Biogen Inc.’s Qalsody (tofersen) for treating amyotrophic lateral sclerosis (ALS), there are still hurdles for the drug to clear, including a confirmatory study and setting a price. Because the U.S. FDA granted Qalsody accelerated, not full approval, there are plenty more data to collect in the ongoing confirmatory phase III Atlas study of those who develop ALS symptoms during the trial compared to placebo. The randomized, double-blind, placebo-controlled study is being conducted with those carrying the superoxide dismutase 1 gene genetic mutation but are symptom free.
A twisted, uncertain path has led to a U.S. FDA approval for Biogen Inc.’s Qalsody (tofersen), the first drug targeting a genetic cause of amyotrophic lateral sclerosis (ALS). Qalsody is for ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene. Patients with SOD-1 mutations account for 2% of ALS cases.
Tofersen’s development is progressing in fits and starts. That was evident at the U.S. FDA’s March 22 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee, which unanimously agreed Biogen Inc.’s failed phase III study predicted a clinical benefit in treating amyotophic lateral sclerosis (ALS) that includes the rare superoxide dismutase 1 component.
Despite a failed phase III study, the U.S. FDA suggests in briefing documents that tofersen (BIIB-067) is effective for treating the rare, genetic disease superoxide dismutase 1 amyotrophic lateral sclerosis (ALS). The intrathecally injected therapy is being developing by Biogen Inc. and Ionis Pharmaceuticals Inc. and is at the heart of a March 22 meeting of the agency’s Peripheral and Central Nervous System Drugs Advisory Committee (adcom).
The U.S. Veterans Health Administration (VA) is being applauded for doing what Medicare has refused to do – provide coverage for Leqembi (lecanemab) in the early stages of Alzheimer’s disease. Under the March 13 VA decision, the Eisai Co. Ltd. drug, which was partnered with Biogen Inc. and granted accelerated approval in January, will be listed as a nonformulary therapy that must be prescribed by a VA-board certified neurologist, geriatric psychiatrist or geriatrician who specializes in treating dementia.
As broader markets struggled throughout 2022, the biopharma industry’s largest and most lucrative companies ended the year in a strong position, according to BioWorld’s Biopharmaceutical Index (BBI), which showed the 22 component stocks climbed by 17.3% throughout the year. Neither the Nasdaq Biotechnology Index (NBI) nor the Dow Jones Industrial Average (DJIA) demonstrated such gains. They were down by 10.9% and 8.8%, respectively. All three indices, however, are following the same path in 2023, shooting up throughout the month of January and back down in recent weeks.
Despite pleas from patient advocacy groups and bipartisan pressure from the U.S. Congress, the Centers for Medicare & Medicaid Services (CMS) isn’t budging on its national coverage determination for amyloid-targeting monoclonal antibodies approved to treat Alzheimer’s.
Now that the U.S. FDA has granted accelerated approval for Biogen Inc./Eisai Co. Ltd.’s early Alzheimer’s drug, Leqembi (lecanemab), the Centers for Medicare & Medicaid Services (CMS) is being pressured to rethink its coverage of amyloid-targeting monoclonal antibodies.
Innocare Pharma Ltd. said it is open to new collaborations after partner Biogen Inc. decided to terminate a global deal to develop and commercialize BTK inhibitor orelabrutinib, in development for multiple sclerosis and other autoimmune diseases. Notice of the termination, disclosed in Biogen’s fourth-quarter 2022 financial report, was “based on the contract term of ‘terminate for convenience,’” Innocare told BioWorld. “We do not know their internal decision-making process.”
Woebot Labs Inc. posted uplifting news for women who have suffered from baby blues and their big brother, post-partum depression. The company recently enrolled the first patient in its pivotal trial of WB001, a digital therapeutic for post-partum depression (PPD). WB001 received U.S. FDA breakthrough device designation in 2021, indicating the acute need for accessible treatment for the common condition.
