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news.The European Commission (EC) has granted orphan drug designation (ODD) to Medipal and JCR Pharmaceuticals' investigational drug, JR-446, to treat mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B.
The therapy is presently under investigation in a Phase I/II trial in Japan. Credit: Julia Koblitz/Unsplash.
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This follows a similar endorsement by the US Food and Drug Administration (FDA).
Sanfilippo syndrome type B is an autosomal recessive disease marked by mutations in the N-acetyl-alpha-glucosaminidase (NAGLU) gene.
JR-446 utilises JCR’s J-Brain Cargo technology and indicated promising non-clinical outcomes in mitigating CNS symptoms associated with this complex condition.
This blood-brain barrier-penetrating technology has been developed to bring biotherapeutics into the central nervous system.
Presently under investigation in a Phase I/II trial in Japan (JR-446-101), it represents a collaborative effort between Medipal and JCR.
The ODD from the EC provides multiple incentives designed to expedite JR-446’s development process in the EU.
Additionally, designated drugs are granted market exclusivity for a period of 10 years in the EU, along with scientific guidance.
IZCARGO (pabinafusp alfa), another product developed from this technology platform, has already received approval in Japan for treating a lysosomal storage disorder.
In September 2023, both companies formalised their partnership through a licensing agreement enabling Medipal to market JR-446 across regions outside Japan.
Additionally, Medipal agreed to assist with clinical development efforts within Japan by managing investigational drug distribution, raising disease awareness, and promoting clinical trials.
JCR’s therapies address conditions such as MPS II, growth disorder, Fabry disease, renal anaemia and acute graft-versus-host disease. It is also developing therapies for rare diseases like MPS I, MPS II, MPS IIIA and B.
