Date: 2018-08-07
Type of information: Granting of the orphan status in the US
Product name: WVE-210201
Compound: synthetic stereopure antisense oligonucleotide specific for human dystrophin pre-messenger ribonucleic acid (mRNA)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Action mechanism:
- antisense oligonucleotide. WVE-210201 is an investigational stereopure oligonucleotide that has been shown to induce skipping of exon 51 of dystrophin pre-mRNA in nonclinical studies and is intended for the treatment of Duchenne muscular dystrophy (DMD). Approximately 13% of DMD patients have genetic mutations that are amenable to treatment with exon 51 skipping therapy. Exon-skipping technology has the potential to induce cellular machinery to ‘skip over’ a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional, dystrophin protein. Wave pre-clinical in vitro experiments using gymnotic delivery (free uptake) of WVE-210201 in DMD patient-derived myoblasts demonstrated efficient exon 51 skipping and dystrophin protein restoration. Preclinical Western blot studies of WVE-210201 demonstrated 52% dystrophin protein restoration as compared with normal skeletal muscle tissue lysates.
Company: Wave LIfe Sciences (USA - MA)
Disease: Duchenne muscular dystrophy
Latest news:
- • On August 16, 2018, Wave Life Sciences announced that the FDA has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD).
- WVE-210201 is currently being studied in an ongoing global, multicenter, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in DMD patients with gene mutations amenable to exon 51 skipping. The trial is expected to enroll up to 40 patients, including ambulatory and non-ambulatory patients, between the ages of 5 and 18 years of age. As patients complete the Phase 1 trial, they have the option to enroll in an ongoing open label extension study in which they receive continued treatment with WVE-210201.
- • On July 31, 2018, the European Commission has granted orphan drug designation for WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD).
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2018-08-07
Orphan status UE: 2018-07-31
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
