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Kyowa Kirin Co., Ltd. has successfully completed the acquisition of Orchard Therapeutics.
The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin. The integrated business will now increase its focus on meeting the needs of people living with devastating genetic and other severe diseases where the burden is immense and current treatment options are limited or do not exist.
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. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. 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 U.S., where it is currently an investigational drug under Priority Review by the Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.
Using the same hematopoietic stem cell (HSC) gene therapy technology platform, Orchard Therapeutics is also progressing two clinical-stage programs, 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.
“We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy. 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, Ph.D., 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.”
“We look forward to this next chapter in Orchard Therapeutics’ evolution and are eager to partner with our new colleagues at Kyowa Kirin to unlock the full potential of our HSC gene therapy approach,“ said Bobby Gaspar, co-founder and chief executive officer of Orchard Therapeutics. “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 U.S., 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 program in MPS-IIIA and earlier-stage research programs.”
In the new organisational structure, Bobby Gaspar, M.D., Ph.D., will report to Kyowa Kirin President & CEO Masashi Miyamoto, Ph.D., and become a member of the Kyowa Kirin's senior R&D leadership team, helping the organization evaluate next-generation therapeutic candidates. Members of the Orchard Therapeutics team will continue to operate from its existing facilities in London and Boston. Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.