The FDA granted rusfertide breakthrough remedy designation for sufferers with polycythemia vera.
An investigational drug known as rusfertide might quickly supply a brand new remedy choice for individuals dwelling with polycythemia vera, a uncommon blood most cancers that causes the physique to provide too many pink blood cells. Protagonist Therapeutics introduced that the U.S. Meals and Drug Administration (FDA) has granted rusfertide breakthrough remedy designation, a step that might velocity its path to approval.
This designation provides to rusfertide’s earlier orphan drug and quick observe statuses, all of which offer regulatory benefits designed to assist new therapies attain sufferers extra rapidly. Within the information launch, Protagonist mentioned it plans to file a brand new drug utility with the FDA within the fourth quarter of 2025.
Rusfertide is a first-in-class remedy that acts as a hepcidin-mimetic peptide. In polycythemia vera, excessive pink blood cell counts result in thickened blood, which will increase the chance of blood clots, stroke and different critical problems. Normal care typically contains drugs akin to hydroxyurea, interferon or ruxolitinib, together with common phlebotomies (blood attracts) to maintain hematocrit ranges beneath management. Nevertheless, many sufferers proceed to wrestle with uncontrolled blood counts, remedy uncomfortable side effects, and disease-related fatigue.
By mimicking hepcidin, a pure hormone that regulates iron within the physique, rusfertide helps cut back extra pink blood cell manufacturing. In medical trials, the remedy has proven the flexibility to manage hematocrit ranges, cut back the necessity for frequent phlebotomies and enhance signs akin to fatigue.
Optimistic Outcomes from Part 3 VERIFY Trial of Rusfertide in Polycythemia Vera
The FDA’s choice was supported by outcomes from the worldwide section 3 VERIFY trial, which enrolled 293 sufferers with polycythemia vera. VERIFY is designed as a three-part, placebo-controlled examine lasting 156 weeks, testing rusfertide in sufferers who stay depending on phlebotomy regardless of customary remedy.
On the 2025 American Society of Scientific Oncology (ASCO) Annual Assembly, 32-week knowledge from VERIFY have been introduced throughout a plenary session, underscoring the drug’s potential to alter remedy follow.
In accordance with the information launch from Protagonist, sufferers who obtained rusfertide plus their present remedy achieved sturdy outcomes throughout each major and secondary trial finish factors. These included dependable hematocrit management, fewer phlebotomy procedures and significant enhancements in patient-reported outcomes akin to power and fatigue ranges.
“We’re very happy with the FDA’s choice, which highlights rusfertide’s potential to display substantial enchancment over accessible therapies,” mentioned Dr. Dinesh V. Patel, president and CEO of Protagonist Therapeutics, within the information launch. “We stay on observe to submit our NDA by the tip of this yr.”
Dr. Arturo Molina, chief medical officer at Protagonist, added within the information launch, “The excellent knowledge present compelling proof that rusfertide might assist sufferers with polycythemia vera who can not obtain satisfactory hematocrit management with customary remedies.”
FDA Designations and What They Imply for Rusfertide in Polycythemia Vera
Breakthrough remedy designation is granted to experimental remedies that present early proof of offering a big profit over present choices for critical situations. It makes a remedy eligible for nearer FDA steerage, quicker growth timelines and precedence evaluate.
Orphan Drug standing, which rusfertide obtained in 2020, is given to medicines concentrating on uncommon illnesses. It may possibly present extra advantages akin to market exclusivity and tax incentives. Quick observe designation helps velocity the FDA evaluate course of. Collectively, these designations place rusfertide for an accelerated path towards potential approval.
All sufferers from the managed portion of the VERIFY examine have now moved into the open-label extension, the place researchers are persevering with to trace long-term security and efficacy.
If accredited, rusfertide may develop into the primary remedy of its type for polycythemia vera. For sufferers who rely closely on phlebotomy or wrestle to keep up secure hematocrit ranges on present medication, rusfertide might characterize a brand new choice that immediately targets the underlying biology of the illness.
References
- “Rusfertide Receives Breakthrough Remedy Designation for Therapy of Erythrocytosis in Sufferers with Polycythemia Vera,” by Protagonist Therapeutics, Inc. Information launch; Aug. 25, 2025.
- “Rusfertide Elicits Promising Responses in Polycythemia Vera,” by Alex Biese. CURE. Information; March 3, 2025. https://www.curetoday.com/view/rusfertide-elicits-promising-responses-in-polycythemia-vera
- “Rusfertide Reduces Phlebotomy Want, Improves High quality of Life in Polycythemia Vera,” by Ryan Scott. CURE. Information; June 11, 2025. https://www.curetoday.com/view/rusfertide-reduces-phlebotomy-need-improves-quality-of-life-in-polycythemia-vera
- “Increasing on a New Solution to Handle Polycythemia Vera With out Iron Deficiency,” by Dr. Aaron Gerds. CURE. Video; June 10, 2025. https://www.curetoday.com/view/expanding-on-a-new-way-to-manage-polycythemia-vera-without-iron-deficiency
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