Posted 9 August 2019
AbbVie has filed a marketing application with the FDA seeking approval of Orilissa for the management of heavy menstrual bleeding (HMSB) associated with uterine fibroids.
The application is supported by data from a phase 3 program involving almost 800 women at approximately 100 sites in the US and Canada.
Orilissa in combination with add back therapy reduced HMB, measured by the alkaline hematin method compared to placebo.
The FDA approved the drug for the management of endometriosis-associated pain under priority review last year.
First FDA approved therapy for rare joint tumour
Daiichi Sankyo has become the first sponsor to offer a treatment for tenosynovial giant cell (TGC) tumour with its capsule Turalio approved this week in the US.
Turalio is now available for adult patients with symptomatic TGC tumour associated with severe morbidity or functional limitations and not responsive to improvement with surgery.
The approval was based on results from an international trial of 120 patients which found significant improvement in overall response rate (ORR). Patients receiving Turalio demonstrated an ORR of 38 per cent, compared to no responses in patients who received placebo.
The complete response rate was 15 per cent and the partial response rate was 23 per cent.
However, it also bears a boxed warning for the risk of potentially fatal liver injury.
Enzyme deficiency disease gets priority
The has FDA granted Alnylam's marketing application for givosiran priority review for the treatment of acute hepatic porphyria (AHP).
The submission is supported by data from the Envision study that evaluated 94 patients and showed the drug significantly reduced the annualised rate of composite porphyria attacks compared to placebo.
The FDA previously granted givosiran breakthrough therapy and orphan drug designation. An action date has been set for 4 February 2020.
Alynlam has also indicated plans to file a marketing authorisation application to the EMA this year.
Skin cancer breakthrough
The FDA has granted breakthrough designation to BMS and Nektars' investigational new drug NKTR-214 in combination with BMS' Opdivo for the treatment of patients with previously untreated unresectable or metastatic melanoma.
The designation is supported by data from the ongoing PIVOT-02 study investigating 38 patients of whom 53 per cent achieved a complete or partial response at 12.7 months.
The complete response rate was 34 per cent and 42 per cent of patients achieved a maximum reduction of 100 per cent in target lesions.
The companies are currently enrolling patients for a phase 3 clinical trial evaluating efficacy, safety and tolerability.