Date: 2018-08-27
Type of information: Refusal of a Market Autorisation in the US
Product name: Waylivra™
Compound: volanesorsen - phosphorothioate oligonucleotide targeted to apolipoprotein C-III
Therapeutic area: Genetic diseases - Metabolic diseases - Rare diseases
Action mechanism:
- antisense oligonucleotide. Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (ISIS-APOCIIIRxSIS-APOCIIIRx) is an antisense drug that targets apoC-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides.
Company: Isis USA Ltd (UK) Isis Pharmaceuticals (USA - CA) Akcea Therapeutics (USA - MA)
Disease: familial chylomicronaemia syndrome (FCS)
Latest news:
- • On August 27, 2018, Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, and Ionis Pharmaceuticals announced that they received a Complete Response Letter (CRL) from the Division of Metabolism and Endocrinology Products of the FDA regarding the New Drug Application (NDA) for Waylivra™(volanesorsen). Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with Waylivra™ reduced triglycerides by 77% (-94% when compared to placebo). The Endocrine Society and current clinical practice guidelines recommend triglyceride reduction as the goal of treatment for FCS. The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels.
- • On June 23, 2015, the FDA has granted orphan drug designation for volanesorsen sodium, apolipotrotein C-III antisense oligonucleotide for the treatment of familial chylomicronemia syndrome (FCS). In a Phase 2 study published in the NEJM in December 2014, patients with FCS treated with volanesorsen achieved substantial reductions in apoC-III, triglycerides, chylomicrons and apoC-III-associated very low density lipoprotein-cholesterol (VLDL-C) particles. Akcea Therapeutics, a wholly owned subsidiary of Isis Pharmaceuticals, is currently conducting an international multi-center, randomized, double-blind, placebo-controlled Phase 3 study in patients with FCS.
• On January 13, 2014, Isis Pharmaceuticals has announced that the Committee for Orphan Medicinal Products (COMP) has issued a positive opinion for orphan drug designation for ISIS-APOCIIIRx for the treatment of patients with familial chylomicronemia syndrome (FCS). ISIS-APOCIIIRx is a wholly owned drug that Isis has evaluated in a broad Phase 2 program. In these studies, treatment with ISIS-APOCIIIRx substantially lowered triglycerides in patients with very high to extremely high triglycerides with reductions of more than 1,500 mg/dL observed in FCS patients.
- • On 7-9 January 2014, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for phosphorothioate oligonucleotide targeted to apolipoprotein C-III for treatment of familial chylomicronaemia syndrome.
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: 2015-06-23
Orphan status UE: 2014-02-19
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
