预约演示

Two major breakthroughs in Fragile X Syndrome treatments

2024-04-16

孤儿药临床2期临床1期临床结果

Fragile X Syndrome typically affects males more severely compared to females and is a significant cause of inherited intellectual disability and autism. Credit: bangoland via Shutterstock.

Fragile X Syndromeeived approval from the US Food and Drug Administration (FDA) for a Phase II clinical triinherited intellectual disabilityme (Fautismhe same week that the European Commission gave orphan drug status to Shionogi’s zatolmilast (BPN14770).

Spinogenixare genetic condition that causes aFood and Drug Administration (FDA)including learning disabilities anSPG601itiveFragile X Syndrome (FXS)r typically affects males more severely than females and is a significant cause of inherited intellectual disability and autism. These issues arise from the silencing of the Fmr1 gene, leading to impaired BK channel function, which is essential for neuron communication.

FXSnogenix’s SPG601 is a novel small molecule BK channel activator that works by binding learning disabilitiescreascognitive impairment to restore synaptic function. The Phase IIa trial will assess the neurophysiological and clinicinherited intellectual disabilityy pilautismadult men diagnosed with FXS. This trial aims tFmr1termine how effective the dBK channelaltering disease markers and alleviating symptoms compared to a placebo.

SpinogenixnogSPG601tella Sarraf emphasised theBK channel activator BK channelfective and patient-friendly solutions for neurodevelopmental conditions such as FXS: “Like many other conditions, loss of synaptic function remains a key driver of disease, and for that reSPG601we are excited to launch our first US trial to adFXSss this unmet need in FXS.”

This deSpinogenixfollows the 11 April announcement that the European Commission has granted orphan medicinal product designation for ShioFXSi’s FXS drug, zatolmilast, a cAMP-specific 3′,5’ cyclic phosphodiesterase 4D inhibitor. Zatolmilast is developed by Tetra Therapeutics, a Shionogi Group company. Shionogi acquired TetraFXS 2020 as part of its efforts to develop medications for unmet medical needs. Zatolmilast received orphan drug designation for FXS in the US in September 2023.

FDA grants orphan drug status to NovelMed’s PNH treatment

FDAgine seeks approval for high-rNovelMedoblastoma treatment

In a Phase II clinical trial assessing zatolmilast, cognitive assessments using the NIH Toolbox revealed significant benefits in oral reading recognition, picture vocabulary, and cognition crystallised composite score (NCT03569631), in males aged 18 to 45 years. The drug is forecast to generate $64m in sales in 2030, according to GlobalData.

Norgine Norgineata is the parent cohigh-risk neuroblastoma Technology.

There are currently no approved treatments for FXS, and prescribed treatment consists exclusively of off-label drugs that target individual symptoms of the disease, such as SSRIs for depressive symptoms and anxiety, and stimulants that include methylphenidate for hyperactivity, inattention, and impulsivity.

According to a report on the GlobalDatazatolmilastelligence Center, the FXS market across two major markets (US and Germany) was valued at $21.9m in 2020 and is projected to grow at a compound annual growth rate of 7.6% reaching $45.8m by 2030.

更多内容，请访问原始网站

文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。

机构