预约演示
FDA grants RMAT designation to Taysha’s Rett syndrome gene therapy
引进/卖出上市批准基因疗法快速通道
Rett syndrome is characterised by loss of communication, slowing or regression of development, and motor and respiratory impairment. Credit: designer491 via Getty Images.
Rett syndromeherapies has received a regenerative medicine advanced therapy (RMAT) designation for motor and respiratory impairment102 from the US Food and Drug Administration (FDA) to treat Rett syndrome.
Taysha Gene Therapieserapy that involves using an AAV9 virus to deliver the MECP2 gene directly into the central nervous systeTSHA-102at the underlFood and Drug Administration (FDA)e genetic Rett syndromeed by mutations in the MECP2 gene that affects brain development. It primarily occurs in girls and results in severe mental and physical disability.
TSHA-102 designation programme is intended to expedite the development and rMECP2 of regenerative therapies to treat, modify, reverse, or cure a serious conditioRett syndromegenetic disorderMECP2
The FDA’s decision to grant the designation to Taysha is supported by initial results from the ongoing Phase I/II REVEAL clinical trials in adults (NCT05606614), and paediatric (NCT06152237) patients. The FDA reviewed safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across the two trials. Initial safety and efficacy data from the high-dose cohorts from both trials is expected in the second half of 2024, as per a 29 February update.
In OFDAber 2022, Astellas announced plans to make a strategic investment to back the development of Taysha’s development programmes for Rett syndrome and giant axonal neuropathy (GAN). The pharma giant gaiFDA an exclusive option for licensing two clinical-stage programmes of Taysha—TSHA-102 and TSHATSHA-102 the treatment of GAN.
See Also:Vertex PAstellasticals gets grant for treatment of alpha-1 antitrypsin deficiency with compound of formula (i)Rett syndromegiant axonal neuropathy (GAN)TSHA-102TSHA-120
BioCryst Vertex Pharmaceuticalsrant for inhibitors of the coalpha-1 antitrypsin deficiencyalpha-1 antitrypsin disease treatment
Things however did not go to plan as about a year later, Taysha discontinued its TSHA-120 development programme following a Type C meeting with the US Food and Drug Administration (FDA), where the agency suggested the company conduct a placebo-controlled study and do a long-term follow-up trial. Following this, Astellas chose not to exercise its right to obtain an exclusive licence for the drug.
BioCryst Pharmaceuticalsthat it would shift its focus to the TSHA-102 programme. According to GlobalData’s Pharma Intelligence Center, TSHA-102 is forecast to generate $650m in sales in 2030.
GlobalData is the parent company of Pharmaceutical Technology.
In the announcement accompanying the designation, Taysha’Tayshaident and head of TSHA-120mar Nagendran said: “We believe receiving RMAT designation US Food and Drug Administration (FDA)d in Rett syndrome and the therapeutic potential of TSHA-102 to change the treatment paradigm.”Astellas
In April 2023, Acadia commercialised the first US Rett syndroTSHA-102Daybue (trofinetide). The drug deactivPharma Intelligence Centero TSHA-102further neurodegeneration, thus preventing injury-induced seizures. Still, there is currently no disease-modifying treatment that treats the genetic root of the disease.
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