XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

Life Science 90

Life Science FDA Approval Insulin Drugs 90

XTalks

APRIL 15, 2025

Soleno Therapeutics has announced the FDA approval of Vykat XR (diazoxide choline extended-release tablets) for the treatment of hyperphagia an intense, persistent hunger in patients with Prader-Willi syndrome (PWS). The approval marks the first FDA-approved therapy specifically targeting hyperphagia in this rare genetic disorder.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Trials 52

XTalks

SEPTEMBER 17, 2020

Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinical trials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.

Trials 98

Trials Gene Hormones Clinical Trials 98