- Taldefgrobep alfa, in Phase 3 global clinical development for
Spinal Muscular Atrophy, granted EU Orphan Drug Designation in
addition to previously receiving Fast Track and Orphan Drug
Designation in the US
NEW
HAVEN, Conn., July 31,
2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN;
"Biohaven") announced today that it received orphan medicinal
product designation from the European Commission (EC) for
taldefgrobep alfa, a novel anti-myostatin adnectin, for the
treatment of spinal muscular atrophy (SMA). Orphan Designation
highlights the potential for taldefgrobep to deliver significant
benefit for people with SMA and provides regulatory and commercial
incentives including a reduction in regulatory fees associated
with protocol reviews and scientific advice, along with an
additional ten-years of market protection. Biohaven previously
received fast track and orphan drug designation from the FDA for
taldefgrobep in the treatment of SMA.
SMA is a rare, progressively debilitating motor neuron disease
in which development and growth of muscle mass are compromised,
resulting in progressive weakness and muscle atrophy, reduced motor
function, impaired quality of life and often death. Inhibition of
myostatin, a naturally occurring protein that limits skeletal
muscle growth, an important process in healthy muscular
development, is a promising therapeutic strategy for SMA.
Taldefgrobep has the potential to be a novel therapy to enhance
muscle function in SMA by blocking myostatin, when used in
combination with currently available disease modifying therapies
that help preserve motor neurons. Taldefgrobep's novelty in a field
of myostatin inhibitors is based on its unique, dual mechanism of
action. It binds to myostatin to lower overall myostatin levels and
also functions as a receptor antagonist, thereby blocking myostatin
signaling in skeletal muscles.
Irfan Qureshi, MD, Chief Medical
Officer, Biohaven commented, "We are delighted that the
European Commission granted orphan drug designation for
taldefgrobep alfa for the treatment of SMA. Children and adults
living with SMA experience significant muscle weakness and
functional impairments affecting their quality of daily life, and a
substantial unmet medical need persists. We are excited about the
potential for taldefgrobep alfa to improve the lives of
patients and families affected by SMA."
As a leader in innovative trials addressing neurodegenerative
diseases, Biohaven is currently enrolling a Phase 3 clinical trial
of taldefgrobep in SMA: A Study to Evaluate the Efficacy and Safety
of Taldefgrobep Alfa in Participants with Spinal Muscular Atrophy
(RESILIENT) (NCT05337553).
About Taldefgrobep alfa
Taldefgrobep alfa
(also known as BHV2000) is a modified adnectin designed to
specifically bind to myostatin (GDF-8). Taldefgrobep is a fully
human anti-myostatin recombinant protein that lowers free myostatin
and acts as an Activin 2b receptor
antagonist with the myostatin-taldefgrobep complex. Adnectins are
an established proprietary protein therapeutic class based on human
fibronectin, an extracellular protein that is naturally abundant in
human serum.
About Spinal Muscular Atrophy (SMA)
Spinal muscular
atrophy (SMA) is a rare genetic neurodegenerative disorder
characterized by the loss of motor neurons, atrophy of the
voluntary muscles of the limbs and trunk and progressive muscle
weakness that is often fatal and typically diagnosed in young
children. The underlying pathology of SMA is caused by insufficient
production of the SMN (survival of motor neuron) protein, essential
for the survival of motor neurons, and is encoded by two genes,
SMN1 and SMN2. In the U.S., SMA affects approximately 1 in 11,000
births, and about 1 in every 50 Americans is a genetic carrier.
About Biohaven
Biohaven is a global clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of life-changing therapies to treat a broad range
of rare and common diseases. Biohaven's experienced management team
brings with it a track record of delivering new drug approvals for
products for diseases such as migraine, depression, bipolar
disorder and schizophrenia. Biohaven is advancing a pipeline of
therapies for diseases with little or no treatment options,
leveraging its proven drug development capabilities and proprietary
platforms, including Kv7 ion channel modulation for epilepsy and
neuronal hyperexcitability, glutamate modulation for
obsessive-compulsive disorder and spinocerebellar ataxia, myostatin
inhibition for neuromuscular diseases, and brain-penetrant
TYK2/JAK1 inhibition for immune-mediated brain disorders.
Biohaven's portfolio of early- and late-stage product candidates
also includes discovery research programs focused on TRPM3 channel
activation for neuropathic pain, CD-38 antibody recruiting,
bispecific molecules for multiple myeloma, antibody drug conjugates
(ADCs), and targeted extracellular protein degrader platform
technology (MoDEs™ platform) with potential application in
neurological disorders, cancer, and autoimmune diseases. For more
information, visit www.biohaven.com.
Forward-looking Statements
This news release includes
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. The use of certain words,
including "continue", "plan", "will", "believe", "may", "expect",
"anticipate" and similar expressions, is intended to identify
forward-looking statements. Investors are cautioned that any
forward-looking statements, including statements regarding the
future development, timing and potential marketing approval and
commercialization of development candidates are not guarantees of
future performance or results and involve substantial risks and
uncertainties. Actual results, developments and events may differ
materially from those in the forward-looking statements as a result
of various factors including: the expected timing, commencement and
outcomes of Biohaven's planned and ongoing clinical trials; the
timing of planned interactions and filings with the Food and Drug
Administration; the timing and outcome of expected regulatory
filings; complying with applicable U.S. regulatory requirements;
the potential commercialization of Biohaven's product candidates;
the potential for Biohaven's product candidates to be first in
class therapies; and the effectiveness and safety of Biohaven's
product candidates. Additional important factors to be considered
in connection with forward-looking statements are described in
Biohaven's filings with the Securities and Exchange Commission,
including within the sections titled "Risk Factors" and
"Management's Discussion and Analysis of Financial Condition and
Results of Operations". The forward-looking statements are made as
of the date of this new release, and Biohaven does not undertake
any obligation to update any forward-looking statements, whether as
a result of new information, future events or otherwise, except as
required by law.
MoDEs is a trademark of Biohaven Therapeutics Ltd.
Biohaven Contact:
Jennifer
Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
201-248-0741
Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
312-961-2502
View original content to download
multimedia:https://www.prnewswire.com/news-releases/biohavens-taldefgrobep-alfa-receives-eu-orphan-drug-designation-for-spinal-muscular-atrophy-301889263.html
SOURCE Biohaven Ltd.