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

Actor-Director Sathish Kaushik Died of a Heart Attack at the Age of 66March 09, 2023
Soleus Push-Ups: A Laziest Exercise to Lower Blood Sugar LevelsMarch 09, 2023
World Kidney Day 2023: Be Aware of Kidney DiseasesMarch 09, 2023
Sushmita Sen reveals about suffering from a severe heart attack recently and Addison's disease in 2014March 06, 2023
A significant increase in influenza A H3N2 patients that are accompanied by severe coughing and feverMarch 06, 2023
Is AIDS or HIV curable in Ayurveda?March 03, 2023
Artificial Sweetener Erythritol Raises the Possibility of Heart Attack.March 01, 2023
Difference between HIV and AIDS - When to give antiretroviral therapy?March 01, 2023
Zero Discrimination Day: Let's Raise the Voice Against DiscriminationMarch 01, 2023
International Women’s Day 2023 – Embrace EquityFebruary 28, 2023
Brain Tumors - Types, Symptoms and Risk FactorsFebruary 28, 2023
World Rare Disease Day: End Stigma Against Rare Diseases.February 28, 2023
National Science Day 2023 - Global Science for Global WellbeingFebruary 28, 2023
Eating disorders - Types and their complicationsFebruary 27, 2023
The condition that took away the life of Malayalam filmmaker, Joseph ManuFebruary 27, 2023
World NGO Day 2023 - We make a living by what we get, but we make a life by what we giveFebruary 27, 2023
Functions and Problems of SerotoninFebruary 25, 2023
Arthritis management – Tips to manage arthritis effectivelyFebruary 25, 2023
Apple has undertaken a project of no-prick blood sugar monitoring feature for its watch.February 24, 2023
Bird flu – Jharkhand on alert, know its prevention and symptoms in humansFebruary 24, 2023