In a significant development for the healthcare industry, London-based pharmaceutical giant GlaxoSmithKline Plc (GSK.L) has received approval from the U.S. Food and Drug Administration (FDA) for its innovative oral therapy, Momelotinib, to treat anemia in patients suffering from myelofibrosis, a rare and debilitating bone marrow cancer. This long-awaited approval comes after a delay in the decision, as the FDA sought to review additional data earlier this year.
Myelofibrosis is a challenging condition characterized by the proliferation of fibrous tissue in the bone marrow, leading to a range of serious complications, including anemia. The disease is associated with high levels of hepcidin, a protein responsible for regulating the body’s iron usage. Elevated hepcidin levels disrupt the production of blood cells, often causing severe anemia, which, in turn, necessitates frequent blood transfusions to maintain adequate red blood cell counts.
GSK’s Momelotinib, marketed under the brand name Ojjaara, belongs to the same class of treatments as other therapies for myelofibrosis but has a unique mechanism of action. It not only addresses the symptoms of the disease but also focuses on reducing the levels of hepcidin in the body, thus addressing one of the root causes of anemia in myelofibrosis patients.
The FDA’s approval of Momelotinib is a significant milestone in the treatment of myelofibrosis as it covers patients “regardless of prior myelofibrosis therapy,” according to GSK’s official statement. This broad approval means that patients who have exhausted other treatment options now have access to this innovative therapy.
The decision to grant approval for Momelotinib is grounded in robust scientific evidence. Data from a late-stage clinical study demonstrated the therapy’s effectiveness in reducing disease symptoms and, crucially, in decreasing patients’ dependence on blood transfusions when compared to patients treated with an alternative anemia therapy called danazol.
Myelofibrosis is an orphan disease, affecting less than 50,000 patients in the United States, according to government data. GSK’s Momelotinib is poised to become a cornerstone in the treatment of this condition, offering hope to patients who have long struggled with the debilitating symptoms and the challenges posed by frequent blood transfusions. Analysts at Morningstar, including Damien Conover, have estimated that Momelotinib could capture close to 50% of the second-line myelofibrosis market, with projected annual peak sales reaching almost £500 million ($627.45 million).
Conover also pointed out the long-term growth potential for GSK, noting that Momelotinib’s patent protection extends beyond 2033 in key markets. This patent protection ensures that GSK can continue to invest in research and development while providing a reliable supply of this essential therapy to patients worldwide.
In a strategic move to bolster its presence in the cancer treatment market, GSK added Momelotinib to its drug pipeline through the acquisition of cancer drug developer Sierra Oncology for $1.9 billion in the previous year. While GSK’s oncology business accounted for only 2% of its total sales in 2022, the company’s commitment to advancing cancer treatments remains unwavering. However, GSK did face setbacks with two cancer compounds previously touted as potential blockbusters, underscoring the challenges inherent in drug development.
Sierra Oncology originally acquired Momelotinib from Gilead Sciences in 2018 for an initial payment of $3 million, with additional milestone payments of up to $195 million. This drug’s journey from one company to another underscores the collaborative and dynamic nature of pharmaceutical research and development.
The FDA’s approval of Momelotinib represents a significant advancement in the treatment options available for myelofibrosis patients, offering hope for an improved quality of life and reduced reliance on blood transfusions. GSK is now positioned to make a substantial impact in the myelofibrosis market with its innovative therapy, Ojjaara, and will continue its ongoing efforts to expand its presence in the competitive but vital oncology sector.