Human beings have had the most amazing abilities in their back pocket, and yet we haven’t seen anything more significant than our ability to grow on a consistent basis. This progressive approach, on our part, has already got the world to hit upon some huge milestones, with technology appearing as a major member of the stated group. The reason why technology enjoys such an esteemed stature among people is, by and large, predicated upon its skill-set, which ushered us towards a reality that nobody could have ever imagined otherwise. Nevertheless, if we look up close for a second, it will become clear how the whole runner was also very much inspired from the way we applied those skills across a real world environment. The latter component was, in fact, what gave the creation a spectrum-wide presence, and consequentially, kickstarted a tech revolution. Of course, this revolution then went on to scale up the human experience through some outright unique avenues, but even after achieving such a monumental feat, technology will somehow continue to produce the right goods. The same has grown increasingly evident over the recent past, and assuming one new healthcare-themed development shakes out just like we envision, it will only make that trend bigger and better moving forward.
The US Food and Drug Administration (FDA) has formally approved SpringWorks Therapeutics’ drug, Ogsiveo for treating a rare connective tissue tumor, named desmoid tumor. Talk about the condition a little bit, desmoid tumors are essentially related to cancers of connective tissue called sarcomas. Now, while they don’t spread throughout a person’s body like all the other iterations of cancer do, they can be locally aggressive, invading surrounding healthy tissues such as joints, muscle, and internal organs. Such an impact means desmoid tumors are highly likely to cause chronic pain, disfigurement, internal bleeding, and significantly impaired range of motion. As for which population group is at the highest risk of contracting this disease, it is most prevalent in young adults between 20 and30 years of age, showcasing a two-to-three-fold predominance in females. Furthermore, it is estimated that desmoid tumors affect 2 to 4 per million people worldwide, and there are 900 to 1,500 new cases diagnosed per year in the United States. Mind you, though, we have had treatments involving surgical removal of the tumor, but from what we have seen thus far, the approach has been largely unsuccessful in abating a recurrence of cancer. Enter SpringWorks Therapeutics’ latest brainchild. Ogsiveo is a small molecule designed to block an enzyme called gamma secretase, which is known for blocking the activation of Notch receptor. This is a major detail, as nullifying of the stated receptor is what contributes to tumor growth. Having said that, FDA’s nod to the drug only came after an extensive study, where SpringWorks enrolled 142 adults with progressing desmoid tumors not amenable to surgery. Dividing patient into two groups, the study prescribed Ogsiveo to one group and placebo to the other one. The primary goal here was to measure progression-free survival, simply defined as the length of time a patient is alive after the start of treatment and until the tumors progress. Apart from that, the study even made a point to analyze tumor shrinkage. Going by the available details, the results generated along the way showed more improvement across the group that was prescribed the real drug, in comparison to the placebo contingent. To give you concrete numbers, the Ogsiveo managed to put-together a 71% reduction in the risk of disease progression. It also displayed early and sustained improvements in patient-reported outcomes, including pain desmoid tumor-specific symptoms, physical/role functioning, and overall health-related quality of life. Although the drug did, in some cases, birth side-effects like diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, dyspnea etc, they were observed to be manageable to a larger degree.
“Our team is honored to deliver the first FDA-approved therapy for patients with desmoid tumors. This community has been waiting for an effective treatment that not only shrinks their tumors but also significantly improves pain, which is the most debilitating symptom reported by people living with desmoid tumors,” said Saqib Islam, Chief Executive Officer of SpringWorks. “We are pleased with the broad label, which includes all progressing adult patients and specifically references improvement in pain, and believe OGSIVEO has the potential to become the new standard of care for people living with these devastating tumors.”
In case you weren’t aware, Ogsiveo was, at first, a part of Pfizer’s pipeline. However, after advancing the drug till Phase 1 testing, the pharmaceutical giant would hand the keys to its spinout in SpringWorks. As a result of the agreed conditions, though, Pfizer can receive up to $232.5 million in milestone payments, an amount completely separate from the royalties it will receive on sales. Interestingly enough, that’s not the only the only tumor-related drug SpringWorks got from Pfizer, as the former is also working on the development of mirdametinib, which blocks MEK pathway to present a treatment for neurofibromatosis type 1-associated pexiform neurofibromas, or NF1-PN. Make no mistake, MEK inhibitors are already available as cancer treatments, but these drugs aren’t well tolerated by patients. SpringWorks will try and offer clinical efficacy, along with a manageable tolerability profile. The said pursuit is already running on full throttle, given the company reported positive preliminary Phase 2b data. Assuming everything goes according to the plan, SpringWorks will file an FDA submission for mirdametinib in the first half of 2024.
Coming back to Ogsiveo, it is set to be available at a price of $29,000 for a 30-day supply.
“Today is an extraordinary day for the desmoid tumor community. This approval is the culmination of a collaborative effort between the patient community, academia and the biopharmaceutical industry, who worked together with tenacity and persistence to advance promising science,” said Jeanne Whiting, Executive Director Emeritus and Co-Founder of the Desmoid Tumor Research Foundation. “Our hope is that patients and their families will benefit from greater awareness of desmoid tumors, faster diagnoses, and better outcomes now that there is an approved and effective treatment.”
