Source: Rezolute, Inc. 1/23/2024
Rezolute, Inc. (Nasdaq: RZLT), a
clinical-stage biopharmaceutical company committed to developing novel,
transformative therapies for serious metabolic and rare diseases, today
announced that the U.K. Medicines and Healthcare products Regulatory
Agency (MHRA) has awarded the innovative medicine designation, the
Innovation Passport, to RZ358 for the treatment of hypoglycemia due to
congenital hyperinsulinism (HI). The Innovation Passport designation was
granted based on the substantial unmet medical need in this condition
and the potential for RZ358 to benefit patients as evidenced by the
Phase 2 RIZE study in congenital HI, which safely demonstrated
significant improvements in hypoglycemia events.
The Innovation Passport designation in the U.K. is the entry point to the Innovative Licensing and Access Pathway (ILAP). The goal of ILAP is to accelerate the time to market and facilitate patient access to medicines. The Innovation Passport is the first step in the ILAP process, which activates the Medicines and Healthcare products Regulatory Agency (MHRA) and its partner agencies, including the National Institute for Health and Care Excellence (NICE), and the Scottish Medicines Consortium (SMC).
“Congenital hyperinsulinism is the most frequent cause of severe, persistent hypoglycemia in newborn babies, infants and children,” said Susan Stewart, J.D., Chief Regulatory Officer at Rezolute. “The Innovation Passport opens the door for Rezolute to discuss access considerations for potential future indications for RZ358. We are thrilled to receive this designation and work closely with the U.K. and other regulatory authorities to bring this meaningful therapy to patients in need.”
About Congenital Hyperinsulinism
Congenital
hyperinsulinism (congenital HI) is the most common cause of recurrent
and persistent hypoglycemia in children. Patients with congenital HI
typically present with signs or symptoms of hypoglycemia within the
first month of life. These episodes can result in significant brain
injury and death if not recognized and managed appropriately.
Additionally, recurrent, or cumulative, hypoglycemia can lead to
progressive and irreversible damage over time, including serious and
devastating brain injury, seizures, neuro-developmental problems,
feeding difficulties, and significant impact on patient and family
quality of life. In cases of congenital HI that are unresponsive to
medical management, surgical removal of the pancreas may be required. In
those with diffuse congenital HI where the whole pancreas is affected, a
near-total pancreatectomy can be undertaken, although about half of
these children will continue to have hypoglycemia and require medical
treatment for congenital HI.
About RZ358
RZ358
is a fully human monoclonal antibody that works downstream from the
pancreas and instead binds to a unique allosteric site on insulin
receptors in the liver, fat, and muscle. The antibody counteracts the
effects of excess insulin binding and activity, thereby improving
hypoglycemia. Rezolute believes that RZ358 is ideally suited as a
potential therapy for congenital HI and other conditions characterized
by excessive insulin activity (hyperinsulinism). Because RZ358 acts
downstream from the pancreas, it has the potential to be universally
effective at treating congenital HI, regardless of the causative genetic
defect, as well as acquired forms of HI such as those mediated by
insulinomas and other tumor types. RZ358 received Orphan Drug
Designation in the United States and European Union for the treatment of
congenital HI, as well as Pediatric Rare Disease Designation in the
U.S. In the Phase 2 RIZE study, participants with congenital HI ages 2
and older nearly universally achieved significant improvements in
hypoglycemia across multiple endpoints, including the primary and key
secondary endpoints planned for the sunRIZE study. At doses and
exposures that are planned for the Phase 3 study, RZ358 was generally
safe and well-tolerated, and resulted in median improvements in
hypoglycemia exceeding 80%. Based on the RIZE clinical trial outcomes
and the evidence of benefit in this serious condition with substantial
unmet medical need, RZ358 was subsequently granted a priority medicines
(PRIME) designation by the European Medicines Agency (EMA) and an
Innovation Passport designation by the U.K. Innovative Licensing and
Access Pathway (ILAP) Steering Group for the treatment of congenital HI.
Rezolute strives to disrupt current treatment paradigms by developing transformative therapies for devastating rare and chronic metabolic diseases. Its novel therapies hold the potential to both significantly improve outcomes and reduce the treatment burden for patients, treating physicians, and the healthcare system. Rezolute is steadfast in its mission to create profound, positive, and lasting impacts on patients’ lives. Patient, clinician, and advocate voices are integrated in the Company’s drug development process. Rezolute places an emphasis on understanding the patient’s lived experiences, enabling the Company to boldly address a range of severe conditions.
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