GSK’s Ojjaara wins FDA approval for myelofibrosis patients battling anaemia

UNITED KINGDOM — GSK’s myelofibrosis therapy, Ojjaara, also known as momelotinib, has received the coveted stamp of approval from the US Food and Drug Administration (FDA).

This development marks a significant milestone, as Ojjaara becomes the very first treatment option tailored for myelofibrosis patients grappling with the added burden of anaemia.

The FDA’s decision casts a wide net of hope, encompassing both newly diagnosed and previously treated adults who find themselves facing intermediate or high-risk myelofibrosis.

This includes individuals afflicted by primary or secondary myelofibrosis, effectively broadening the treatment horizon for this relentless and rare blood cancer.

Myelofibrosis, a rare and debilitating blood cancer, has long been a medical challenge with limited treatment options.

The ordeal often forces patients into a cycle of transfusions, with more than 30% eventually discontinuing treatment due to the overwhelming burden of anaemia.

At the heart of myelofibrosis lies the Janus kinase (JAK) proteins, notorious for their involvement in inflammation and cancer.

In 2011, Incyte’s Jakafi pioneered the JAK inhibitor treatment for myelofibrosis, followed by Bristol Myers Squibb’s Inrebic in 2019, and CTI Biopharma’s Vonjo in the previous year, cementing the JAK inhibitor’s role in treatment.

Ojjaara, however, takes a distinctive approach. This once-a-day oral small molecule doesn’t merely target two JAK proteins but also A receptor type 1 (ACVR1), also known as activin receptor-like kinase-2 (ALK2).

By blocking this third target, Ojjaara effectively reduces hepcidin levels, a hormone that governs iron usage in the body—a critical aspect in myelofibrosis where hepcidin levels run high, contributing to anaemia.

The FDA’s resounding endorsement of Ojjaara finds its bedrock in the pivotal phase 3 MOMENTUM study.

In this trial, Ojjaara went head-to-head with another anaemia treatment known as danazol, addressing anemic, symptomatic, and JAK inhibitor-experienced myelofibrosis patients.

The results were nothing short of remarkable, with Ojjaara-treated patients experiencing remarkable improvements in symptoms, spleen size, and achieving transfusion independence when compared to their danazol-receiving counterparts.

The efficacy and safety data emanating from a subpopulation of adult patients with anaemia from the late-stage SIMPLIFY-1 trial further fortified GSK’s resounding application for FDA approval.

Ojjaara’s journey has been nothing short of remarkable. Its origin traces back to YM Biosciences, acquired by Gilead Sciences in 2012.

Yet, mixed Phase 3 results under Gilead led to an abrupt halt in its development. Sierra Oncology, however, breathed new life into the program in 2018.

The subsequent Phase 3 study yielded promising outcomes, surpassing Incyte’s Jakafi—a drug that raked in US$2.4 billion in revenue the previous year across its four approved indications.

Following Ojjaara’s Phase 3 triumphs, GSK swiftly moved to secure its future with a US$1.9 billion acquisition of Sierra.

Nina Mojas, Senior Vice President of Oncology Global Product Strategy at GSK, expressed the significance of this milestone by saying, “The vast majority of myelofibrosis patients eventually develop anaemia, causing them to discontinue treatments and require transfusions.

“Given this high unmet need, we are proud to add Ojjaara to our oncology portfolio and address a significant medical need in the community.”

This approval comes hot on the heels of GSK’s commitment to invest over £270 million (US$ 334.5 million) in the construction of a state-of-the-art vaccine manufacturing facility at its Wavre campus in Belgium.

Designed to enhance manufacturing efficiency while reducing environmental impact, this facility is poised to revolutionize the production of freeze-dried vaccines, including GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy.

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