Cemdisiran, a small interfering RNA drug that Regeneron Pharmaceuticals licensed from Alnylam Pharmaceuticals, met the goals of its pivotal test in generalized myasthenia gravis. The rare neuromuscular disease has several new therapies, but analysts see cemdisiran as having advantages over one particular class of drugs.
Precigen’s Papzimeos is the first FDA-approved drug for the rare disease recurrent respiratory papillomatosis, or RRP. While this immunotherapy carries blockbuster expectations, Precigen’s financial reports indicate budgetary challenges.
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The FDA approved Ionis Pharmaceuticals’ Dawnzera for preventing swelling attacks caused by the rare disease hereditary angioedema. A Takeda Pharmaceutical product dominates this market, but Ionis has clinical data showing patients had better outcomes after switching to Dawnzera from Takeda’s drug and other currently available HAE medications.
Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing publicly for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program.
Though anselamimab failed its Phase 3 test in light chain amyloidosis, AstraZeneca points to encouraging results in a pre-defined patient subgroup — which may not be a winning strategy. Prothena resurrected its failed drug candidate for the disease based on a subgroup analysis, only to see that antibody fall short in a pivotal study.
Capricor Therapeutics said the FDA asked for more data supporting the efficacy of Deramiocel, the biotech’s off-the-shelf cell therapy for treating the heart complications of Duchenne muscular dystrophy. Those data could come from an ongoing Phase 3 study expected to yield preliminary results soon.
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KalVista Pharmaceuticals’ Ekterly is now FDA approved for treating acute swelling attacks from hereditary angioedema. The KalVista tablet provides an alternative to injectable or infused HAE medications from companies such as Takeda Pharmaceutical and CSL Behring.
Novartis’s Cosentyx failed to beat a placebo in a Phase 3 clinical trial in giant cell arteritis. The antibody drug was being developed as an alternative to Roche and AbbVie drugs whose approved uses include treating this autoimmune disorder affecting blood vessels.
Neopharmed Gentili is acquiring European rights to Orladeyo, bringing the Italy-based pharmaceutical company into rare disease. BioCryst’s Orladeyo is approved for treating patients with the rare disease hereditary angioedema.
Technology that offers tools that conform, de-identify, link and aggregate data and data science tools like AI, machine learning and advanced analytics, can help those researching rare diseases overcome the hurdles they face in discovery and development.
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Rocket Pharmaceuticals adjusted the clinical trial design to include use of approved drug to avert an excessive immune response to its Danon disease gene therapy. The patient who died was one of two who received this drug under the revised protocol.
Johnson & Johnson’s Imaavy is now FDA-approved for treating generalized myasthenia gravis, introducing competition to Argenx and UCB drugs that treat this rare neuromuscular disorder. J&J is also developing its new drug for other diseases and projects it could reach $5 billion in peak sales across multiple rare and prevalent conditions.
Regulus Therapeutics drug farabursen is a potential first-in-class treatment for a rare kidney disease whose only FDA-approved therapy introduces severe toxicity risks. Farabursen is joining a Novartis drug lineup spanning several therapies for rare renal disorders.
Abeona Therapeutics makes Zevaskyn by engineering a patient’s biopsied skin cells to express the collagen that they lack, then growing those cells into sheets that can be surgically applied to a wound. Abeona's cell-based gene therapy is just the third FDA-approved treatment for epidermolysis bullosa, a rare disease that leads to skin described as thin and fragile like a butterfly’s wings.
Glycomine’s lead program is an experimental treatment for PMM2-CDG, an ultra-rare enzyme deficiency often misdiagnosed as cerebral palsy. With encouraging results from an open-label Phase 2 study, the biotech startup is now preparing for a placebo-controlled test that could potentially support a regulatory submission.