Please login to the form below

Not currently logged in
Email:
Password:

GSK’s application for momelotinib in myelofibrosis with anaemia accepted by FDA

40% of myelofibrosis patients are already anaemic at the time of diagnosis and nearly all patients are estimated to develop anaemia eventually

GSK

GSK’s New Drug Application (NDA) for momelotinib has been accepted by the US Food and Drug Administration (FDA) for patients who have myelofibrosis with anaemia.

The FDA is due to make a decision on momelotinib – which GSK maintains may address a ‘significant unmet need’ – by 16 June 2023. If accepted, the commercial launch of momelotinib is anticipated in 2023.

The NDA is based on results from key phase 3 trials, including the pivotal MOMENTUM trial – a global, randomised, double-blind phase 3 clinical trial of momelotinib versus danazol in patients with myelofibrosis who were symptomatic and anaemic and had been previously treated with an FDA-approved Janus kinase (JAK) inhibitor.

The study met all its primary and key secondary endpoints, demonstrating that momelotinib achieved a statistically significant and clinically meaningful benefit on symptoms, splenic response and anaemia.

Myelofibrosis is a rare blood cancer that results from dysregulated JAK-signal transducer and activator of transcription protein signalling affecting around 20,000 patients in the US, characterised by constitutional symptoms, splenomegaly and progressive anaemia.

Anaemia is the main reason why people with myelofibrosis discontinue treatment, with around 40% of patients already anaemic at the time of diagnosis and nearly all patients estimated to develop anaemia eventually.

Furthermore, currently approved JAK inhibitors only address symptoms and splenomegaly and are myelosuppressive, potentially leading to worsening anaemia and dose reductions that potentially reduce treatment effect.

Momelotinib is a JAK1/2 inhibitor, which treats symptoms of the cancer, but also targets other pathways called ACVR1 and IRAK1 that are thought to protect against anaemia.

Momelotinib was most recently developed by Sierra Oncology – a California-based, late-stage biopharmaceutical company focused on targeted therapies for the treatment of rare cancers – which GSK acquired in July 2022 in a deal worth £1.5bn.

Stephen Dilly, president and chief executive officer, Sierra Oncology, said at the time of the NDA submission: “Today is truly momentous for everyone at Sierra Oncology and the patients we serve. This team designed a targeted study to address the highest unmet need and delivered incredible results in the midst of a pandemic. We are thrilled to submit this NDA on behalf of myelofibrosis patients and look forward to working with the FDA over the coming months.”

Article by
Emily Kimber

18th August 2022

From: Regulatory

Share

Tags

Subscribe to our email news alerts

PMHub

Add my company
Onyx Health

Onyx Health is a healthcare communications and PR agency based in the North East of England, but with a national...

Latest intelligence

Rare diseases: not so rare after all
The brave new world addressing rare diseases – a way ahead to better and more inclusive treatment...
Webinar: Evolving Patient Journeys - How to generate deeper insights in a changing healthcare landscape
Register now for part one in a series, Evolving Patient Journeys, in which our experts look at the main ways in which the patient journey has evolved in recent years....
DIGITAL EXPERIENCES: LEARNINGS FROM OUTSIDE PHARMA
It’s long been the case that pharma lags behind other industries when it comes to digital adoption and engagement. So, what can we learn from these other industries, and how...