Rilzabrutinib granted FDA Fast Track Designation for treatment of immune thrombocytopenia
November 18 2020 - 1:15AM
Rilzabrutinib granted FDA Fast Track Designation for
treatment of immune thrombocytopenia
- Phase 3 trial initiated to evaluate rilzabrutinib, the
potential first BTK inhibitor (Bruton’s tyrosine kinase inhibitor)
for the treatment of immune thrombocytopenia
- Rilzabrutinib previously granted FDA orphan drug
designation
PARIS – November 18, 2020 – 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 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.
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Sanofi is transforming scientific innovation into healthcare
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