BioSpace

UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease

Kygevvi is indicated for patients with thymidine kinase 2 deficiency whose symptoms arise by 12 years of age. The disease ...

FDA Approves KYGEVVI® (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare an…

This novel therapy offers the first FDA-approved option to address the root cause of this ultra-rare condition, which causes ...
STAT

BridgeBio study data on drug for rare form of muscular dystrophy could set up approval

BridgeBio said an experimental therapy for a rare form of muscular dystrophy succeeded in a key trial, potentially setting up ...
Fierce Pharma

On a roll, UCB picks up another rare disease FDA approval

UCB has scored an FDA approval for ultra-rare disease treatment Kygevvi, continuing the positive momentum for the Belgian ...
GlobalData on MSN

FDA approves UCB’s Kygevvi for genetic mitochondrial disease

The approval was based on data from a Phase II trial, two retrospective chart review studies and an expanded access programme ...
Seeking Alpha

Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping

SAN DIEGO, Feb. 20, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates ...
EconoTimes

Novartis to Acquire Avidity Biosciences for $12 Billion to Strengthen Rare Muscle Disorder Portfolio

Swiss pharmaceutical giant Novartis has announced a definitive agreement to acquire U.S.-based biotech firm Avidity ...
pharmaphorum

UCB bags FDA okay for first drug to treat rare disease TK2d

That research led to the FDA granting compassionate use of the doxecitine and doxribtimine treatment in Arturito Estopinan, ...

'We don’t have time': Parents of 4-year-old with rare disorder urge FDA to reconsider drug denial

The FDA denied approving DCA to treat a rare disorder called PDCD. The Higbees are urging the agency to reconsider.
Seeking Alpha

Avidity Biosciences Honors Rare Disease Day®

SAN DIEGO, Feb. 29, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates ...