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.

The post Avidity Bio’s RNA Therapy Flashes Early Signs of Treating a Muscular Dystrophy’s Root Cause appeared first on MedCity News.

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.

The post Ipsen, Genfit Drug That Missed in MASH Wins FDA Approval in a Much Rarer Liver Disease appeared first on MedCity News.

To win the battle against rare diseases, drug developers must work hand-in-hand with patients.

The post Allying With Patients to Make Every Cure Possible appeared first on MedCity News.

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.

The post Drug Licensed from Sanofi Becomes First FDA-Approved Therapy for Ultra-Rare Primary Immunodeficiency appeared first on MedCity News.

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 post Vertex Pharma Bolsters Immunology Profile With $4.9B Alpine Immune Sciences Acquisition appeared first on MedCity News.

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.

The post AstraZeneca’s Rare Disease Pipeline Notches Another FDA Approval appeared first on MedCity News.

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.

The post FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease appeared first on MedCity News.

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.

The post AstraZeneca Acquisition Brings Drug to Fill a Rare Disease Gap Left by Takeda appeared first on MedCity News.

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.

The post Takeda Puts Up $300M to Partner on Protagonist Drug for Rare Blood Disease appeared first on MedCity News.

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 post Ionis Rare Disease Drug Is Headed to FDA, But Forthcoming Data Will Decide Competitive Profile appeared first on MedCity News.

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 post Gearing Up to Challenge Pfizer in a Rare Disease, BridgeBio Secures Up to $1.25B appeared first on MedCity News.

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.

The post Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease appeared first on MedCity News.

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.

The post Chiesi Rare Skin Disease Drug Acquired in Billion Dollar Deal Wins FDA Approval appeared first on MedCity News.

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 post With More 2024 FDA Filings Planned, Novartis Drug Starts Showing Blockbuster Potential appeared first on MedCity News.

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.

The post FDA Approves Not One, But Two Sickle Cell Disease Gene Therapies appeared first on MedCity News.

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.

The post Novartis Nabs FDA Nod for Rare Disease Drug Rival to AstraZeneca Meds appeared first on MedCity News.

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.

The post FDA Approval of SpringWorks Drug Is the First for Rare Connective Tissue Tumor appeared first on MedCity News.

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.

The post Takeda Drug Wins First FDA Approval in Rare, Inherited Blood Clotting Disorder appeared first on MedCity News.

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.

The post New AstraZeneca, Cellectis Alliance Spans Up to 10 Cell & Gene Therapies appeared first on MedCity News.

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.

The post Sarepta Gene Therapy Misses in Phase 3; Prospects Now Rely on FDA Flexibility appeared first on MedCity News.

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.

The post Novartis Drug From $3.2B Deal Succeeds in Phase 3, 2024 FDA Filing Is Planned appeared first on MedCity News.

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.

The post MedCity Pivot Podcast: Tackling Rare Disease With Someone Who Was Touched By It appeared first on MedCity News.

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.

The post Biogen Bulks Up in Rare Disease With $7.3B Reata Pharmaceuticals Acquisition appeared first on MedCity News.

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.

The post FDA Refuses to Review Biohaven Drug for Rare Neuromuscular Disease With No Treatments appeared first on MedCity News.

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.

The post Here’s Why Novartis Is Paying $500M to Acquire a Preclinical RNA Startup appeared first on MedCity News.

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 post RA Capital Leads $150M Financing for Septerna and Lead Drug With Oral Edge in Rare Hormone Disease appeared first on MedCity News.

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.

The post UCB Muscle Disease Drug Wins FDA Nod on Heels of Rival’s Regulatory Approval appeared first on MedCity News.

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.

The post Ipsen Itching Drug Scratches Off a Second FDA Approval in Rare Liver Indication appeared first on MedCity News.

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.

The post Novartis Lines Up $3.2B Deal for Kidney Disease Drugs, But FTC Questions Loom appeared first on MedCity News.

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.

The post FDA Approves First Drug for Rare Skin Disorder, and It’s a Redosable Gene Therapy appeared first on MedCity News.