Kyowa goes big on Libmeldy with $477m Orchard buy

As Libmeldy awaits US FDA approval, Kyowa Kirin acquires Orchard Therapeutics with an aim of maximizing the value of the gene therapy.

Under the terms of the deal, Japan-based pharmaceutical firm, Kyowa will pay $477 million to acquire Orchard. According to Kyowa, the decision to acquire Orchard is spurred by the ongoing and future launches of gene therapy Libmeldy (atidarsagene autotemcel), which is used to treat children with metachromatic leukodystrophy (MLD).

Libmeldy is an ex vivo Hematopoietic Stem Cell (HSC) gene therapy and the European Medicine Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended approval for the one-time treatment in October 2020. In December 2020, the European Commission (EC) granted Orchard approval for Libmeldy.

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Kyowa said the acquisition will enable it to “maximize the value” of Libmeldy.The deal comes at a time where the gene therapy is gunder Priority Review by the US Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) aim date of March 2024.

In addition, Kyowa said it will advance the development of OTL-203 and OTL-201, which use the same HSC gene therapy technology platform as Libmeldy. The two clinical-stage programs aim to treat mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH), mucopolysaccharidosis type IIIA (MPS-IIIA), and paediatric neurometabolic disorders.

“With this transaction, we anticipate being able to use a new modality that can have a profound impact on patients’ lives,” said Takeyoshi Yamashita, director of the board, chief medical officer, senior managing executive officer of Kyowa Kirin.

“Orchard Therapeutics is a company with a steady track record in this field and has already launched its HSC gene therapy in Europe and filed for review in the US. Our hope is to combine the strengths of Kyowa Kirin and Orchard Therapeutics with mutual respect to realize the successful creation and delivery of life-changing value for patients living with rare and life-threatening inherited diseases.”

Kyowa will combine Orchard’s HSC gene therapy platform technology with its own capabilities, infrastructure, and resources. In turn, the firm said it will continue to accelerate various biopharmaceutical candidates, including indications for oncology and autoimmune diseases.

“This is an exciting opportunity designed to accelerate the realization of our shared vision of ending the devastation caused by severe genetic diseases and deliver life-changing value in medical care,” said Bobby Gaspar, CEO of Orchard Therapeutics.

“We remain as true to our mission as ever, and joining Kyowa Kirin’s global network ensures we are well-resourced to progress anticipated commercialization of [Libmeldy] in the US if approved, continue investing in initiatives aimed at accelerating Libmeldy growth in Europe, capitalize on opportunities for global expansion, as well as advance our next-in-line neurometabolic programs in MPS disorders and earlier-stage research programs. We look forward to collaborating with our new colleagues at Kyowa Kirin to fully unlock the curative potential of HSC gene therapy for the benefit of patients and society.”