Syros receives FDA orphan drug designation for Tamibarotene for the treatment of MDS

Latest FDA news

Syros Pharmaceuticals, a leader in the development of medicines that control the expression of genes, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to tamibarotene for the treatment of myelodysplastic syndrome (MDS). Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with azacitidine in the SELECT-MDS-1 Phase 3 trial for RARA-positive patients with newly diagnosed higher-risk MDS (HR-MDS).

“The FDA’s orphan drug designation is an important milestone in the development of tamibarotene as a treatment for MDS,” said David A. Roth, M.D., Syros’ Chief Medical Officer. “We believe tamibarotene’s novel mechanism of action, promising clinical activity data, oral delivery, and favorable tolerability profile supports a potential new option for the approximately 30% of HR-MDS patients who are RARA-positive. We are focused on developing the first potential therapy for a targeted population in HR-MDS as we continue to advance our ongoing SELECT-MDS-1 pivotal trial.”

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

The ongoing SELECT-MDS-1 Phase 3 clinical trial is evaluating the safety and efficacy of tamibarotene in combination with azacitidine for RARA-positive patients with newly diagnosed HR-MDS. Data from the pivotal trial are expected in the fourth quarter of 2023 or the first quarter of 2024, with a potential new drug application filing expected in 2024.

Syros is also evaluating tamibarotene in combination with azacitidine and venetoclax for RARA-positive patients with newly diagnosed unfit acute myeloid leukemia (AML), for which tamibarotene had previously received orphan drug designation. Safety lead-in data from the ongoing SELECT-AML-1 Phase 2 trial is expected in the second half of this year.

Tags : #syros #Tamibarotene #Venetoclax #MDS #TreatmentofMDS

About the Author


Team Medicircle

Related Stories

Loading Please wait...

-Advertisements-




Trending Now

Exploring the Link Between Covid-19 Vaccination and Sudden Cardiac Arrests: ICMR StudyNovember 22, 2023
Powering the Future: China's Biodegradable Wireless System for Bioelectronic InnovationNovember 22, 2023
IMS BHU Pioneers Advanced Cardiac Care: Unveiling Cutting-Edge Technologies for Precise DiagnosisNovember 18, 2023
Inito's $6 Million Boost: Transforming Women's Health with AI-Backed Fertility MonitoringNovember 18, 2023
Madras High Court Advocates for Equality: PG Medical Students to Receive Incentive Marks for COVID-19 DutyNovember 18, 2023
Next-Gen Healthcare: CarePods and AI Redefine the Patient ExperienceNovember 17, 2023
Air Quality Crisis: Alarming Rise in Premature Births Linked to Air Quality in Begusarai, BiharNovember 17, 2023
Limerick Contest-2023: A Celebration of Wit and PoetryNovember 17, 2023
IIIT-Hyderabad among top 100 institutions for 5G Use Case LabNovember 17, 2023
Powerful, motivating and inspiring talks marked TEDx Hyderabad Women 2023 which was held with the theme "Two Steps ForwardNovember 17, 2023
Speaking the Diagnosis: How AI in Voice Analysis Is Revolutionizing Diabetes DetectionNovember 17, 2023
FDA Approves Zepbound: A New Medication for Weight Loss by Eli LillyNovember 17, 2023
CoverSelf Raises $8.2 Million in Seed Funding to Revolutionize Healthcare Claims with Innovative Fintech PlatformNovember 16, 2023
From Miracle to Medic: The Extraordinary Journey of India's First Paediatric Liver Transplant RecipientNovember 16, 2023
Combatting Chikungunya: Valneva's Chikungunya Vaccine Cleared by USFDA, Eyes India LaunchNovember 16, 2023
Global Corporate Summit 2023 with the theme Unlocking Opportunities in a Dynamic World heldNovember 16, 2023
2nd IHub-Data Mobility Summit heldNovember 16, 2023
Revolutionary AI-powered technology detects multiple abdominal pathologies simultaneouslyNovember 16, 2023
1 Out of 3 Stroke Patients Suffer Long-Term Health Complications, Says NeurologistNovember 16, 2023
Amrita Hospital, Kochi, Launches App for People with Swallowing DifficultiesNovember 15, 2023