Market Engagement for Onasemnogene Abeparvovec (Zolgensma®) Service & Onasemnogene Abeparvovec Intrathecal (OAV101 IT) Service

The key objective of the Market Engagement Exercise is to seek feedback from the market regarding levels of interest, capability and capacity in relation to delivering the onasemnogene abeparvovec (zolgensma®) service and the onasemnogene abeparvovec intrathecal (OAV101 IT) service. The commissioner isinterested in receiving feedback from the market on their capability and capacity to deliver services for zolgensma® or combined (zolgensma® and OAV101 IT).

It is also anticipated that the findings from this market engagement exercise will assist in the decision-making for the approach to commissioning the Service(s). If, as a result of the market engagement, competition is absent or minimal, in any of the potential Lots/Regions, the Commissioner may use the Most Suitable Provider Process or Direct Award Process C.

Where competition is absent, Direct Award Process A may be utilised. If, as a result of the market engagement, competition is apparent in a potential Lot/Region , a Competitive Process may be undertaken to award the contract for that Lot.

Service Summary: Spinal muscular atrophy, or SMA, is a rare genetic disorder that causes muscle weakness and progressive loss of movement. The severity of SMA can vary greatly between individuals.

Historically, the condition has been classified into different types, determined by clinicians at the time of diagnosis. This classification is based on both the age at which symptoms first appear, and the highest level of motor function achieved by the patient.

The types range from type 0, the most severe form, where symptoms are present before birth and most affected infants don’t survive past 6 months, through to type 4, which develops in adulthood and is associated with mild motor impairment and a normal life span. However, because the disease presents so variably, consistently categorising patients into these types at the point of diagnosis can be challenging.

Onasemnogene abeparvovec (zolgensma®) is a single-use gene replacement therapy made of a viral vector that has been modified to contain the primary gene for the human survival motor neuron (SMN) protein, which is lacking or mutated in people with SMA. When injected, the vector is expected to carry the gene into the nerve cells, enabling production of sufficient amounts of SMN.

It is administered intravenously. Onasemnogene abeparvovec intrathecal (OAV101 IT) is a one-time gene therapy for patients with SMA, proposed to treat people with SMA aged 2 years and over.

This intrathecal injection will be used to treat a distinct population from the indication for Zolgensma®. Following market engagement and review of capable providers, the Commissioner will consider the most appropriate PSR process(es) to formally commission each service.

Interested parties may respond to this Market Engagement Exercise in partnership with other organisations as part of a consortium, subject to them appointing a consortium lead to act on their behalf as the lead contracting party would take overall responsibility for delivery of the service(s) in accordance with the requirements of any future contract(s) if successful. Atamis Project reference C410884.