FDA approval of Novartis’s Fabhalta makes the drug the first approved oral therapy for rare blood disorder paroxysmal nocturnal hemoglobinuria. With clinical data showing superiority versus two infused AstraZeneca drugs, Novartis’s pill is well-positioned to take market share from those blockbuster products.
SpringWorks Therapeutics drug Ogsiveo is now FDA-approved for treating desmoid tumors, a type of tumor affecting connective tissue. The regulatory decision makes the pill the first therapy approved specifically for treating this rare disorder.
This eBook, in collaboration with Care Logistics, details how hospitals and health systems can facilitate more effective decision-making by operationalizing elevated awareness.
Takeda Pharmaceutical enzyme replacement drug Adzynma is the first FDA-approved therapy for a rare blood disorder affecting fewer than 1,000 Americans. Our regulatory recap also includes the much-anticipated approval of an Eli Lilly weight loss drug and a crackdown on what the FTC says are improperly listed pharma product patents.
AstraZeneca starts the Cellectis partnership with $105 million in upfront cash and an equity investment. The pharmaceutical giant will also cover research costs for up to 10 cell therapy programs in oncology, immunology, and rare diseases.
Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to all patients who have the muscle-wasting disease. Analysts say prospects of the therapy, Elevidys, rest on FDA willingness to exercise flexibility it has already shown to rare disease drugmakers, including Sarepta.
Novartis drug candidate atrasentan met the main goal of a pivotal study in the chronic kidney disease IgA nepropathy. It’s one of two drugs added via the multi-billion dollar acquisition of Chinook Therapeutics; together with a homegrown therapeutic candidate, they give Novartis three different approaches to treat the rare disorder.
In an era of escalating healthcare costs and a growing preference for natural, holistic approaches to health, The Impact Brands emerges as a collective of diverse brands dedicated to supporting overall wellness through natural means.
Rich Horgan believes that there's an alternative path to drug development that can lower costs and the time it takes to bring drugs to market. He is trying to apply it to the field of rare and ultra-rare diseases.
Biogen's Reata Pharmaceuticals acquisition brings Skyclarys, the first and only FDA-approved therapy for the rare neuromuscular disease Friedreich’s ataxia. Biogen says Skyclarys complements the other neuromuscular drugs in its portfolio.
Biohaven’s bid to bring to market the first drug for rare neuromuscular disorder spinocerebellar ataxia has hit a setback. The FDA is refusing to review the drug’s application, a decision that could indicate the agency wants another clinical trial.
Novartis’s acquisition of DTx Pharma brings a lead program for a rare disease with no FDA-approved therapies. But the deal could also help the pharma giant bring new RNA therapies to new places in the body, a top Novartis executive explained.
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.
Septerna Therapeutics’ Series B financing will support plans to reach the clinic with a pill for a rare disease whose only approved therapy is a Takeda Pharmaceutical drug that’s leaving the market. Potential rival therapies are all injectables.
The FDA approved UCB’s Rystiggo for treating generalized myasthenia gravis. Drugs are already available for the rare neuromuscular disorder, but Rystiggo’s approval includes treatment of a subset of patients not addressed by other products.
Ipsen drug Bylvay is now FDA approved for treating pruritus, or severe itching, which is a complication of the rare liver disease Alagille syndrome. The oral drug was previously approved for treating pruritus in another rare inherited liver disease called PFIC.
Novartis is acquiring Chinook Therapeutics in a $3.2 billion deal that brings two late-stage drug candidates for a rare kidney disorder. The Novartis drug pipeline already has a molecule in late-stage development for the same kidney disease, which could raise questions from antitrust regulators.
Krystal Biotech’s Vyjuvek is approved to treat recessive and dominant forms of dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. The redosable gene therapy, applied to the skin, delivers a gene that gets cells to produce collagen that DEB patients lack.