Washington, DC – Over the last few years, we’ve witnessed a significant shift in the regulatory space for rare disease therapeutics, with the FDA and other authorities making conscious efforts to implement much-needed regulatory flexibility to accommodate the practical challenges of trial design for these conditions. For small biotechs and...
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Washington, DC – Despite having not yet received full approval, Travere Therapeutics’ first-in-class endothelin and angiotensin II receptor antagonist Filspari—which received the FDA’s accelerated approval in February 2023—is already making strides in treating IgA nephropathy. A rare autoimmune disease, IgA nephropathy occurs when immunoglobulin A accumulates in the kidneys, leading...
New York, NY – Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials. The last decade has fundamentally changed how clinicians treat spinal muscular atrophy. In 2016, Biogen’s Spinraza became the FDA-approved treatment for SMA,...
Washington, DC – The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published Wednesday in the Journal of the American...
SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say. Every drug approval is a monumental occasion worthy of celebration. But for SpringWorks Therapeutics, the approval...
San Diego, Calif. – The American Association for Cancer Research’s annual conference kicks off Friday as more than 20,000 attendees descend on San Diego. Analysts and researchers who spoke with BioSpace said they’re looking forward to early scientific data, and antibody-drug conjugates (ADCs) will be a key focus. Stay tuned to...
Basel, Switzerland – The past two years have seen a pair of new treatments approved for a particularly intractable neurodegenerative disease—amyotrophic lateral sclerosis. But while Amylyx’s Relyvrio and Biogen’s Qalsody have crossed the FDA finish line, others have stumbled in a regulatory space that experts say is still evolving. The...
Multicenter study sponsored by SELNET to evaluate cirtuvivint as a second-line monotherapy in selected sarcoma subtypes SAN DIEGO, Calif. — Biosplice Therapeutics, Inc. (“Biosplice”), a clinical-stage biotechnology company pioneering advancements in small molecule inhibition of CDC-like kinases (CLK) and Dual-specificity tyrosine phosphorylation-regulated (DYRK) kinases, today announced that the first...
SAN DIEGO, Calif. — Biosplice Therapeutics, Inc. (“Biosplice”), a clinical-stage biotechnology company pioneering advancements in small molecule inhibition of CDC-like kinases (CLK) and Dual-specificity tyrosine phosphorylation-regulated (DYRK) kinases, today announced that the first patient has been dosed in a Phase 1 clinical trial of cirtuvivint in patients with acute myeloid leukemia...
BASEL, Switzerland – BioVersys AG (SIX: BIOV), a multi-asset, clinical stage biopharmaceutical company focusing on research and development of novel antibacterial products for serious life-threatening infections caused by multidrug-resistant (MDR) bacteria, announced today that the European Medicines Agency Committee for Orphan Medicinal Products (EMA COMP) has granted orphan designation for...