Avidity Biosciences’ RNA therapy for facioscapulohumeral muscular dystrophy has preliminary clinical data showing it knocked down expression of a gene that causes this rare disease. More details will be presented at a medical conference this week.
Iqirvo failed as a treatment for the fatty liver disease MASH, but the drug is now FDA approved in primary biliary cholangitis. An Intercept Pharmaceuticals drug already treats this rare liver disease and Gilead Sciences is poised to compete with its PBC drug approaching an FDA decision this summer.
Canada has a proud history of achievement in the areas of science and technology, and the field of biomanufacturing and life sciences is no exception.
To win the battle against rare diseases, drug developers must work hand-in-hand with patients.
The FDA approved X4 Pharmaceuticals drug Xolremdi for treating WHIM syndrome. The molecule, which addresses the underlying cause of this rare immunodeficiency, was licensed from Sanofi’s Genzyme subsidiary.
Vertex Pharmaceuticals gains a Phase 3-ready Alpine Immune Sciences drug with potential applications in a wide range of immunological disorders. The lead indication for the Alpine drug, povetacicept, is the chronic kidney disease IgA nephropathy.
The FDA approved AstraZeneca drug Voydeya as an add-on to standard therapies for paroxysmal nocturnal hemoglobinuria, a rare blood disorder. Alexion, now AstraZeneca’s rare disease subsidiary, added the small molecule to its pipeline via a 2019 acquisition.
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Corticosteroids are a standard first-line treatment for Duchenne muscular dystrophy, but these drugs have many side effects. FDA approval of Italfarmaco’s Duvyzat is the first nonsteroidal drug to pass the agency’s regulatory bar for treating this rare disease.
AstraZeneca is acquiring Amolyt Pharma to get eneboparatide, a drug in Phase 3 development for treating hypoparathyroidism. Takeda Pharmaceutical markets the only FDA-approved treatment for this rare disease, but the Japanese pharmaceutical giant plans to stop making the drug.
Protagonist Therapeutics' rusfertide could have preliminary data next year from its Phase 3 test in polycythemia vera. If the peptide drug wins FDA approval in this rare blood disorder, the partners will share in U.S. commercialization while Takeda is responsible for the drug in the rest of the world.
In its Phase 3 test, Ionis Pharmaceuticals drug donidalorsen reduced the frequency of swelling attacks caused by the rare disease hereditary angioedema. If Ionis can commercialize this drug, competition would include products from Takeda Pharmaceutical and BioCryst Pharmaceuticals.
The TSX Venture Exchange has a strong history of helping early-stage health and life sciences companies raise patient capital for research and development.
BridgeBio will receive $500 million in exchange for royalties from sales of acoramidis, a drug currently under FDA review for treating heart complications caused by the rare disease transthyretin amyloidosis. The deal also refinances lending agreements, providing BridgeBio with access to additional capital.
The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.
Chiesi Group’s Filsuvez is the second drug to win FDA approval for epidermolysis bullosa and the first for junctional EB, a more severe form of the rare skin disease. The birch tree bark-derived drug is from Chiesi’s $1.25 billion Amryt Pharma acquisition.
Novartis drug iptacopan, which won its first FDA approval in early December in a rare blood disorder, has met the main goal of a pivotal test in an ultra-rare kidney disease. The small molecule’s potential to address many diseases has stirred up blockbuster expectations.
The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.