KalVista Pharmaceuticals brings to Chiesi Group the product Ekterly, the first FDA-approved oral drug for acute treatment of swelling attacks from the rare disease hereditary angioedema. Ekterly’s tablet formulation offers a dosing edge compared to injectable HAE medications.

The post Chiesi Group Grows Again in Rare Disease With $1.9B KalVista Acquisition appeared first on MedCity News.

Intellia Therapeutics’ lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to the swelling attacks from the rare disease hereditary angioedema. Intellia has filed a regulatory submission for this genetic medicine, which could become the first in vivo gene-editing therapy to land FDA approval.

The post Intellia’s Data Reveal Tees Up FDA Filing for CRISPR-Based In Vivo Gene-Editing Med appeared first on MedCity News.

Travere Therapeutics’ Filspari is now the first FDA-approved drug for focal segmental glomerulosclerosis (FSGS), a rare kidney disease. Analysts project the pill will become a blockbuster seller.

The post Bouncing Back From Trial Failure, Travere Wins First FDA Approval in Rare Kidney Disease appeared first on MedCity News.

Biogen’s Apellis Pharmaceuticals acquisition comes nearly two years after the drugmaker purchased immunology startup Human Immunology Biosciences. CEO Chris Viehbacher said Apellis accelerates Biogen’s expansion in nephrology, supporting a HI-Bio drug currently in pivotal testing for three kidney conditions.

The post Why Biogen Is Paying $5.6B to Buy Apellis Pharma appeared first on MedCity News.

Rocket Pharmaceuticals received FDA approval for Kresladi, a gene therapy developed to treat leukocyte-adhesion deficiency type 1, an inherited immunodeficiency that can become fatal to infants and young children. Kresladi is the first gene therapy for this ultra-rare disease and the first commercial product for Rocket.

The post FDA Approval Makes Rocket Pharma Gene Therapy the First for Ultra-Rare Immune Disorder appeared first on MedCity News.

Denali Therapeutics’ Avlayah received FDA approval for treating Hunter syndrome, a rare neurological disorder. The biologic medicine uses Denali’s proprietary drug delivery technology to cross the protective blood-brain barrier.

The post FDA Approval for Denali Therapeutics Blazes a New Trail for Brain-Penetrating Drugs appeared first on MedCity News.

Leucovorin is now approved for cerebral folate deficiency months after FDA Commissioner Marty Makary claimed the decades-old generic drug had promise for treating autism. The FDA’s review was based on published literature and real-world evidence.

The post FDA Drug Approval Marks a First for a Disease — But It’s Not Autism appeared first on MedCity News.

Povetacicept, a Vertex Pharmaceuticals immunology drug designed to block two targets, met the main goal of a Phase 3 clinical trial in the rare kidney disease immunoglobulin A nephropathy. The fusion protein came from Vertex’s acquisition of Alpine Immune Sciences, and analysts say the drug has best-in-class potential.

The post Vertex Pharma to Seek Speedy FDA Approval for Kidney Disease Drug From $4.9B Acquisition appeared first on MedCity News.

GSK licensed to Alfasigma global rights to linerixibat, a drug developed to treat the rare liver disease primary biliary cholangitis (PBC). The move follows Alfasigma’s 2025 voluntary market withdrawal of Ocaliva, a PBC drug that had sparked safety concerns from U.S. and European regulators.

The post Alfasigma Pays $300M for Rights to GSK Rare Liver Disease Drug On Track for FDA Decision appeared first on MedCity News.

The Series B extension brings Atavistik Bio’s Series B financing to $160 million. The startup’s lead program is in development for hereditary hemorrhagic telangiectasia (HHT), an inherited bleeding disorder.

The post Startup Atavistik Adds $40M for Clinical Trial in Rare Bleeding Disorder With No Approved Therapies appeared first on MedCity News.

Ampreloxetine’s Phase 3 failure in multiple system atrophy is the third disappointing clinical trial outcome for the Theravance Biopharma drug. The biotech will now wind down all R&D and explore options that could include the sale of the company.

The post Theravance Drug Fails Another Pivotal Test, This Time Sparking Restructuring That Ends R&D appeared first on MedCity News.

Atrium spun out of Avidity Biosciences, which Novartis acquired in a $12 billion deal. The Atrium pipeline is led by two RNA therapies with a path to the clinic for rare inherited cardiological disorders that have no FDA-approved drugs.

The post Atrium Therapeutics Launches With $270M for RNA Therapies Addressing Rare Cardio Conditions appeared first on MedCity News.

It’s not just about awareness but also a call to action for patients, families, employers, payers and policymakers to modernize the systems that shape access to care. It must focus on connecting innovation with the infrastructure that governs approval, coverage and affordability.

The post Rare Disease Day 2026: From Awareness to Access in a Volatile Year for Patients and Specialized Therapies appeared first on MedCity News.

The FDA asked for more data to determine whether Disc Medicine’s bitopertin is benefiting patients with erythropoietic protoporphyria, a rare blood disorder. Analysts say it’s a surprising delay considering bitopertin is one of the first drugs in a new FDA pilot program that speeds up regulatory review of medicines deemed critical for national security or public health.

The post Disc Medicine Rare Disease Drug Picked for Faster Regulatory Review Is Rejected by the FDA appeared first on MedCity News.

PTC Therapeutics said it cannot resolve differences it has with the FDA in interpreting clinical trial data for Translarna, a drug it developed for Duchenne muscular dystrophy. The rare disease drug developer’s decision to withdraw Translarna’s FDA submission comes nearly a year after a negative European regulatory decision for the drug.

The post PTC Therapeutics Ends Bid to Seek FDA Approval for Rare Muscle Disease Drug appeared first on MedCity News.

Infigratinib achieved statistically significant improvement in growth rate and body composition in a Phase 3 clinical trial that tested the BridgeBio Pharma drug in achondroplasia, the most common form of dwarfism. Regulatory submissions are planned for later this year.

The post BridgeBio Stands Tall as Phase 3 Data Put Dwarfism Drug on Track for FDA Filing appeared first on MedCity News.

Brepocitinib is on track for pivotal testing after meeting the goals of a proof-of-concept study in cutaneous sarcoidosis, a rare inflammatory skin disorder with no FDA-approved therapies. Roivant Sciences subsidiary Priovant Therapeutics licensed the dual JAK1 and TYK2 inhibitor from Pfizer.

The post Clinical Trial Win Bolsters Blockbuster Prospects for Roivant Rare Disease Drug Licensed From Pfizer appeared first on MedCity News.

The Digital Medicine Society (DiME) launched a new set of FDA-aligned digital measures to standardize how outcomes are tracked in pediatric rare disease trials. The framework aims to make trials faster and more efficient, giving researchers and drugmakers a practical roadmap for developing new therapies for children who often have no effective treatment options.

The post How DiME’s New Pediatric Rare Disease Project Could Make Clinical Trials Faster appeared first on MedCity News.

Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe.

The post Clinical Trial Holds Cast Doubt on Approval Chances of Regenxbio Gene Therapy for Rare Disease appeared first on MedCity News.

Zenas Biopharma’s obexelimab achieved a statistically significant and clinically meaningful reduction in signs and symptoms of immunoglobulin G4-related disease, but not to the extent shown by Amgen’s Uplizna in its pivotal study. Still, Zenas points to features of its drug that could make it competitive as a maintenance therapy for the rare autoimmune disorder.

The post Zenas Bio Inflammation Drug Meets Phase 3 Goals, But Is It Enough to Compete With Amgen? appeared first on MedCity News.

Omeros’s complement system drug Yartemlea is now FDA approved for treating a severe and potentially fatal complication of hematopoietic stem cell transplants. Our recap of other regulatory actions includes drug approvals in cancer, rare disease, and metabolic disorders.

The post Fatal Complication of Stem Cell Transplants Gets Its First FDA-Approved Therapy appeared first on MedCity News.

BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics is a marriage of two rare disease biotech companies. Amicus’s two commercialized products, for the enzyme deficiencies Fabry disease and Pompe disease, are each projected to become blockbuster sellers.

The post BioMarin’s Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition appeared first on MedCity News.

Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology.

The post FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder appeared first on MedCity News.

Protagonist Therapeutics’ drug rusfertide reduced the need for phlebotomies as a way to treat the rare blood cancer polycythemia vera. Longer-term Phase 3 data for the Takeda Pharmaceutical-partnered peptide drug were presented during the annual meeting of the American Society of Hematology.

The post Shining Light on How Protagonist, Takeda Drug Brings Treatment of a Rare Blood Disease Out of the Dark Ages appeared first on MedCity News.

Pharvaris’s deucrictibant met the main and secondary goals of its Phase 3 test as an on-demand treatment for swelling attacks caused by the rare disease hereditary angioedema. Pharvaris’s capsule could compete against Kalvista Pharmaceuticals’ Ekterly, an HAE pill approved by the FDA over the summer.

The post Pharvaris Drug for Rare Swelling Disease Meets Phase 3 Goals; FDA Filing Planned for 2026 appeared first on MedCity News.

Regeneron Pharmaceuticals will share in development of Tessera Therapeutics’ TSRA-196, a Phase 1-ready gene-editing therapy for alpha-1 antitrypsin deficiency (AATD). Other companies developing genetic medicines for this rare disease include Beam Therapeutics, Wave Life Sciences, Korro Bio, and AIRNA.

The post Regeneron Puts Up $150M to Partner on Tessera Gene-Editing Med for Rare Liver & Lung Disorder appeared first on MedCity News.

Protego Biopharma’s lead program is a potential treatment for the rare disease light chain amyloidosis. The startup says the novel mechanism of its oral small molecule should have better outcomes than antibodies from AstraZeneca and Prothena that failed their respective pivotal studies in the disorder earlier this year.

The post Startup Protego Bio Lands $130M for First-in-Class Drug’s Pivotal Test in Rare Plasma Disorder appeared first on MedCity News.

Arrowhead Pharmaceuticals’ RNAi drug plozasiran, brand name Redemplo, is now the second FDA-approved therapy for familial chylomicronemia syndrome, an inherited lipid disorder. Beyond its claims of safety and dosing advantages, Arrowhead set a dramatically lower price for its first commercial product, which will compete against an Ionis Pharmaceuticals drug.

The post With FDA Approval for RNAi Drug, Arrowhead Pharma Starts Price War With Rival Ionis appeared first on MedCity News.

Eli Lilly is paying $75 million up front for global rights to AAV-AIPL1, a gene therapy on track for U.S. and European regulatory submissions in the rare, inherited eye disorder Leber congenital amaurosis 4, or LCA4. The deal brings to Lilly another late-stage gene therapy for the eyes following the pharma giant’s October acquisition of Adverum Biotechnologies and its lead program for wet AMD.

The post Eli Lilly Strikes Another Genetic Meds Deal, Gaining Global Rights to MeiraGTx Retinal Gene Therapy appeared first on MedCity News.

The six new drugs awarded FDA Commissioner’s National Priority Vouchers brings to 15 the total number of therapies selected for the new pilot program. Under this program, the FDA says the standard 10- to 12-month review time could be shaved down to “within months.”

The post FDA Adds 6 More Meds to Pilot Program for Speedy Review of Drugs in the National Interest appeared first on MedCity News.