Orchard will help Kyowa Kirin grow in gene therapy

Japan’s Kyowa Kirin has reached an agreement to buy Orchard Therapeutics of the UK in a deal valued at up to $478 million, ahead of an FDA decision on the biotech’s lead gene therapy.

The $16-per-share deal has an upfront value of around $387 million, coupled with a contingent value right of $1 per share, tied to the US approval of Libmeldy (atidarsagene autotemcel), Orchard’s gene therapy for metachromatic leukodystrophy (MLD), a rare hereditary disease that causes damage to peripheral and central nerves.

Libmeldy was approved in Europe in December 2020 and involves harvesting haematopoietic stem cells (HSCs) from the bone marrow or blood of patients, modifying them to replace the defective ARSA gene associated with the disorder with a functional version, and then re-introducing them into the body in a single intravenous infusion.

It was filed for approval in the US in August, and the company has been granted a priority review by the FDA for the therapy, known in the US as OTL-200, with a decision from the regulator due by 18th March next year.

Libmeldy, which costs £2.8 million (around $3.7 million), is reimbursable in several European countries, including the UK and Germany, and made sales of $6.6 million in its most recent quarter, taking the total generated from the therapy in Europe to $26 million since launch.

A July report from the Institute for Clinical and Economic Review (ICER) in the US states that the therapy could be cost-effective at a price between $2.3 million and $3.9 million, and has a chance of “filling the promise of possible treatments for conditions that previously were untreatable.”

Kyowa Kirin said taking over Orchard would give it a leadership position in “the burgeoning field of genetic medicine”, with a pipeline of commercial, clinical, and preclinical therapies that all draw on the biotech’s HSC approach.

Following after Libmeldy are OTL-203 and OTL-201 for the treatment of another group of severe paediatric neurometabolic disorders, mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome), respectively.

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

“Our hope is to combine the strengths of Kyowa Kirin and Orchard Therapeutics with mutual respect to realise the successful creation and delivery of life-changing value for patients living with rare and life-threatening inherited diseases.”