Rilzabrutinib granted FDA Fast Track Designation for treatment of immune thrombocytopenia.

▴ Rilzabrutinib granted FDA Fast Track Designation for treatment of immune thrombocytopenia.
The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD), for the treatment of immune thrombocytopenia.

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to the oral investigational Bruton’s tyrosine kinase (BTK) inhibitor, rilzabrutinib, which has the potential to be the first BTK inhibitor for the treatment of immune thrombocytopenia (ITP). In addition, following positive Phase 1/2 study results, a Phase 3 study evaluating rilzabrutinib for ITP has been initiated. Rilzabrutinib received orphan drug designation from the FDA for the treatment of ITP in October 2018.

"By awarding Fast Track Designation to rilzabrutinib, an investigational candidate for the treatment of ITP, the FDA has recognized rilzabrutinib's potential to meaningfully improve outcomes for patients with this debilitating disease. This is an excellent acknowledgement as we initiate our Phase 3 study,” said Dolca Thomas, Chief Medical Officer of Principia, a Sanofi company. “FTD is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.”

About Fast Track Designation

FTD is an FDA process designed to facilitate the development, and expedite the review of, medicines to treat serious conditions and fill an unmet medical need. The FDA created this process to help deliver important new drugs to patients earlier, and it covers a broad range of serious illnesses. Fast Track designation can lead to an Accelerated Approval and Priority Review if certain criteria are met.

About Immune thrombocytopenia

ITP is characterized by immune-mediated platelet destruction and impairment of platelet production, which leads to downstream thrombocytopenia, a predisposition to bleeding, and adverse impact on patient quality of life. There remains an unmet medical need in ITP to achieve rapid and durable remission rates for patients who have relapsed or are refractory to corticosteroids.

About Rilzabrutinib

Rilzabrutinib is an oral, reversible covalent, Bruton’s tyrosine kinase (BTK) inhibitor being investigated for the treatment of immune-mediated diseases.  BTK is involved in innate and adaptive immune responses and is a signalling molecule in immune-mediated diseases. Rilzabrutinib data demonstrate an ability to block inflammatory immune cells, eliminate autoantibody destructive signalling, and prevent new autoantibody production without depleting B cells. Rilzabrutinib has the potential to target the underlying disease pathogenesis and has not been shown to alter platelet aggregation. The clinical significance of these mechanisms is currently under investigation and its safety and efficacy have not been reviewed by any regulatory authority.

Editor’s Note: Rilzabrutinib is being investigated in a Phase 3 trial for pemphigus, an immune-mediated disease characterized by blisters in mucous membranes and skin.  Additionally, a Phase 2 study in the autoimmune condition IgG4 Disease has also been initiated.

Tags : #US #FDA #FastTrackDesignation #Sanofi #Treatment #ImmuneThrombocytopenia #rilzabrutinib

Related Stories

Loading Please wait...

-Advertisements-




Trending Now

HR leaders ready to shape India's future workforce - XLRI and Leadup Universe seminar in PuneNovember 05, 2024
Nestlé India and SM Sehgal Foundation celebrate 5 years of Project Vriddhi November 05, 2024
Why Tuberculosis Is Still the World’s Leading Killer in 2024 And How We’re Failing to Stop ItNovember 05, 2024
Crompton Launches the Nigella Pro Mixer Grinder to Revolutionize Kitchen ConvenienceNovember 05, 2024
Invisible Shadows: The Growing Crisis of Corneal Blindness in IndiaNovember 05, 2024
Thai AirAsia Entrusts Çelebi India with Ground Handling for New Hyderabad- Bangkok Flights, Signalling Further India Expansion November 05, 2024
Thai AirAsia Entrusts Çelebi India with Ground Handling for New Hyderabad- Bangkok Flights, Signalling Further India Expansion November 05, 2024
Discover 5 Platforms That Turn Your Pennies into PowerhousesNovember 04, 2024
Breathless in the Capital: Post-Diwali Smog Triggers Health Alarms in DelhiNovember 04, 2024
Is Your Diet Fuelling Your Disease? The Shocking Link Between Oil and Multiple SclerosisNovember 04, 2024
Unmasking the Danger Within: The Alarming Rise of Stomach Cancer in IndiaNovember 04, 2024
Hospital-acquired infections are fuelling antimicrobial resistance October 30, 2024
Transforming Patient Care: HealthTech Startups Making Waves in HealthcareOctober 30, 2024
Hospital-acquired infections are fuelling antimicrobial resistance October 30, 2024
Impressive Growth & Margin Expansion 237% YoY Surge in Net ProfitsOctober 29, 2024
Ramaiah Memorial Hospital Announces the Launch of its Comprehensive State-of-the-art Ramaiah Stroke Center on World Stroke Day October 29, 2024
CarDekho Group's New Auto Segment’s Festive Trend Report: SUVs Lead the Way with a 43% YoY Traffic Increase Across All Sub-SegmentsOctober 29, 2024
World Stroke Day by Dr Praveen Gupta, Principle Director and Chief of Neurology, Fortis Hospital.October 29, 2024
HighTide Therapeutics to Present Analyses of Phase 2 MASH Study at AASLD's The Liver Meeting 2024October 29, 2024
AI Gone Wild? Why OpenAI’s Whisper - Transcription Tool used in Hospitals Might Be Putting Lives at RiskOctober 29, 2024