Let children with dwarfism grow 9 cm tall! Positive phase 2 clinical data of biomarin

Today, biomarin announced that its developing drug vosoritide has significantly improved the growth of children with achondroplasia in a phase 2 clinical trial Vosoritide increased the average patient's natural history by 9 cm over a 54 month study Achondroplasia is the most common type of short limb dwarfism, which is an autosomal dominant genetic disease Globally, it affects the lives of 250000 people The disease can lead to serious cardiovascular, neurological and metabolic complications Achondroplasia is caused by mutations in the gene encoding fibroblast growth factor receptor 3 (FGFR3) FGFR3 is a negative regulator of bone development, which can affect the proliferation, activation and differentiation of chondrocytes At present, there is no approved treatment for this disease ▲ multiple serious complications caused by achondroplasia (photo source: biomarin website) Biosoritide (bmn111) developed by biomarin is a C-type natriuretic peptide (CNP) analogue derived from natural human peptides Natural human peptide is a positive regulator of bone growth and development Vosoritide inhibits the over active FGFR3 signaling pathway by binding to specific receptors Previously, the U.S FDA and the European drug administration have granted vosoritide the orphan drug qualification to treat patients with achondroplasia Action mechanism of vosoritide (picture source: Florence large et al [public domain]) Patients aged 5 to 14 years with achondroplasia participated in the phase 2 clinical study The results showed that compared with the height data of patients with natural history, vosoritide increased the average height of patients in the 54 month study by 9 cm, reaching a statistically significant difference In addition, in the past 12 months alone, patients who received vooritide treatment grew 2.2 cm, further proving the potential of vooritide treatment "Our natural history database has about 13000 height related data points By comparing the data of patients participating in the trial with the data of patients with natural history, we get more information supporting this treatment, proving that vosoritide can significantly and continuously improve the growth of patients, "hank, global R & D president of biomarin company "The data from this trial, together with the results of our phase 3 clinical trials, will serve as the basis for future regulatory applications," Fuchs, MD, said Biomarin is expected to release the data obtained by vosoritide in phase 3 trials by the end of this year We are looking forward to more good news for patients from biomarin! reference material: [1] BioMarin Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study， Retrieved November 14, 2019, from https://investors.biomarin.com/2019-11-14-BioMarin-Announces-Cumulative-Additional-Height-Gain-of-9-0-cm-over-54-months-versus-Natural-History-in-Children-with-Achondroplasia-Treated-with-Vosoritide-in-Phase-2-Study [2] BioMarin Official Website，Retrieved November 14, 2019, from https:// A kind of