FDA Approves First Gene Therapy for Treatment of Aromatic L …
1 day ago · The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric ...
FDA approves Kebilidi as first gene therapy to treat aromatic L …
1 day ago · The first gene therapy for for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency for adults and pediatric patients, eladocagene exuparvovec-tneq (Kebilidi; PTC Therapeutics), has been approved by the FDA according to an announcement from the federal agency. 1 AADC deficiency is a rare genetic disorder that affects production of neurotransmitters that are chemical ...
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
13 hours ago · The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. Now, seven years and many approvals later, innovation in the space has hit new heights with ...
PTC Therapeutics Announces FDA Approval of AADC Deficiency …
About KEBILIDI™ (eladocagene exuparvovec-tneq) KEBILIDI is a recombinant adeno-associated virus serotype 2 (rAAV2)-based gene therapy, containing the human DDC gene. It is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.
PTC wins US approval of gene therapy for fatal enzyme disorder
13 hours ago · The approval is welcome news for PTC, which has struggled with a number of setbacks that dented its share price. PTC stock climbed above $58 in May 2023, ... The FDA clearance for Kebilidi is also conditional, using the FDA’s accelerated approval pathway. Regulators are allowing the therapy on the market based on a study that showed early ...
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated
13 hours ago · The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with AADC deficiency. ... News release. PTC Therapeutics ...
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets ...
1 day ago · The FDA on Wednesday signed off on PTC Therapeutics’ gene therapy eladocagene exuparvovec-tneq for the treatment of aromatic L-amino acid decarboxylase deficiency. The treatment will be marketed under the brand name Kebilidi. With only around 350 cases reported in the literature, aromatic L-amino acid decarboxylase (AADC) deficiency is an ultrarare genetic disease characterized by severe ...
US FDA approves PTC Therapeutics' gene therapy for ultra-rare …
1 day ago · The therapy, sold under the brand name Kebilidi, is a one-time treatment for children and adults with an ultra rare inherited disorder called aromatic l-amino acid decarboxylase (AADC) deficiency.
FDA Approves Eladocagene Exuparvovec-Tneq for Treatment of …
15 hours ago · The FDA approved eladocagene exuparvovec-tneq (Kebilidi; PTC Therapeutics Inc) for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Eladocagene exuparvovec-tneq is an adeno-associated virus vector-based gene therapy and became the first FDA-approved gene therapy for AADC deficiency. FDA ...
FDA grants accelerated approval for PTC’s AADC deficiency gene …
16 hours ago · Credit: Gatot Adri via Shutterstock. The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics ’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain. This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency.
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