Japan-based pharma company Kyowa Kirin has successfully completed the acquisition of gene therapy developer Orchard Therapeutics.
Orchard Therapeutics’ portfolio comprises Libmeldy (atidarsagene autotemcel), which is intended for the treatment of eligible patients with early-onset metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system.
Libmeldy is approved by the European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA). Libmeldy is known as OTL-200 in the US, where it is currently an investigational drug under Priority Review by the Food and Drug Administration (FDA).
Using the same haematopoietic stem cell (HSC) gene therapy technology platform, Orchard Therapeutics is also progressing two clinical stage programmes, OTL-203 for the treatment of mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 in development for mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome.
“This platform offers significant potential to deliver more innovative treatments and breakthrough therapies and aligns with our purpose to deliver life-changing value for people living with rare and complex diseases,” said Masashi Miyamoto, Representative Director, President and CEO of Kyowa Kirin. “Going forward, our companies will build on the extensive experience of Orchard’s gene therapy platform and apply it to under-served indications and diseases where we believe it to be scientifically and clinically differentiated.”
Bobby Gaspar, Co-Founder and Chief Executive Officer of Orchard Therapeutics, added: “The next 12 months have the potential to provide several breakout opportunities that we believe would cement our leadership position in the field, including the potential approval and launch of OTL-200 in the US, the acceleration of Libmeldy growth in Europe, the progression of our global registrational trial for OTL-203 in MPS-IH, as well as the advancement of our next-in-line neurometabolic programme in MPS-IIIA and earlier-stage research programmes.”