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On January 27, 2026, the FDA approved daratumumab and hyaluronidase-fihj (Darzalex Faspro; Janssen Biotech) in combination with bortezomib (Velcade; Takeda), lenalidomide (Revlimid; Celgene Corporation), and dexamethasone (decadron; Amneal; quadruplet regimen; D-VRd) for the treatment of adults with newly diagnosed multiple myeloma (NDMM) who are ineligible for autologous stem cell transplant (ASCT).1 This milestone extends the use of subcutaneous daratumumab in a quadruplet frontline regimen, underscoring advances in therapeutic options for patients with NDMM who cannot undergo ASCT.1
Regimen Background and Clinical Context
Table of Contents
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-daratumumab-and-hyaluronidase-fihj-bortezomib-lenalidomide-and-dexamethasone-newly
FDA approves daratumumab and hyaluronidase-fihj for multiple myeloma. FDA. Published May 1,2020. Accessed January 27,2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-daratumumab-and-hyaluronidase-fihj-multiple-myeloma
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An investigational drug, tofersen, has shown promise in slowing the progression of amyotrophic lateral sclerosis (ALS) in a Phase 3 clinical trial, as initially reported in Nature Medicine in 2025. As of January 27, 2026, the drug has received FDA approval for the treatment of ALS patients with a specific genetic mutation.
Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord,causing loss of muscle control. It typically affects both upper and lower motor neurons, leading to paralysis and eventually death. The disease’s progression and symptoms vary substantially between individuals.
Approximately 5-10% of ALS cases are familial, meaning they are inherited, while the remaining 90-95% are sporadic, with no known cause. The average age of onset is between 55 and 75 years, and life expectancy after diagnosis is typically 2-5 years, though some individuals may live much longer.
example: In 2022, approximately 30,000 Americans were living with ALS, according to the ALS Association.
Tofersen and its Mechanism of Action
Tofersen is an antisense oligonucleotide designed to reduce the production of mutant SOD1 protein in patients with ALS caused by mutations in the SOD1 gene.it effectively works by binding to the messenger RNA (mRNA) that carries the genetic code for the SOD1 protein, leading to its degradation and reducing the amount of the harmful protein produced.
The Phase 3 clinical trial, known as VALOR, investigated tofersen in 162 participants with ALS who carried a SOD1 mutation. participants were randomly assigned to receive either tofersen or placebo via intrathecal injection (injection into the spinal fluid) over a 48-week period.
Evidence: The VALOR trial demonstrated a statistically notable slowing of disease progression, as measured by the Revised Amyotrophic Lateral Sclerosis functional Rating Scale (ALSFRS-R), in the tofersen group compared to the placebo group. Specifically, the tofersen group showed a 30% slowing of functional decline, as reported in the Nature Medicine publication.
FDA approval and future outlook
On April 25, 2024, the U.S. Food and Drug Administration (FDA) approved tofersen (Qalsody) for the treatment of ALS in adults with a confirmed SOD1 mutation. This marks the first approved gene therapy for ALS.
The approval was based on data from the VALOR trial and represents a significant advancement in the treatment of ALS, particularly for the subset of patients with SOD1 mutations. The FDA granted tofersen Priority Review and Fast Track designation, recognizing the urgent need for new therapies for this devastating disease.
Example: The FDA approval requires post-marketing studies to further evaluate the long-term effects and benefits of tofersen. Further information on the approval and post-marketing requirements can be found on the FDA website.
- Biogen: The pharmaceutical company that developed and markets tofersen.
- FDA (U.S. Food and Drug Administration): The regulatory agency responsible for approving drugs for use in the United States.
- ALS Association: A non-profit organization dedicated to supporting ALS research and patient care. ALS Association Website
- National Institute of Neurological Disorders and Stroke (NINDS): A component of the National Institutes of Health (NIH) that conducts and supports research on neurological disorders, including ALS. NINDS Website
