Special Edition: Reviewing the advanced therapy pipeline

Santen looks to ways to reduce hurdles for patients in accessing advanced therapies

By Jane Byrne contact

- Last updated on GMT

© GettyImages/Zarina Lukash
© GettyImages/Zarina Lukash

Related tags: Eye, Ophthalmology, Cell therapy

As a relatively new player in the field of cell therapy, Santen Pharmaceutical has outlined how it is looking to collaborate with key stakeholders in order to shape relevant policies, forge meaningful partnerships and create an optimal environment for the launch of an eye disorder focused therapy.

In April 2020, Santen entered the cell therapy field, having negotiated a $252m ex-US licensing deal for jCyte Inc's human retinal progenitor cell (hRPC) injectable therapy. 

The treatment is initially aimed at treating retinitis pigmentosa, a rare vision-limiting disorder endured by nearly 1.9m people globally.

We spoke to the Japanese firm to get a sense of its objectives as it navigates the advanced therapies domain. 

BioPharma-Reporter: How have you been working towards realizing this goal of broader industry collaboration?

Santen:​ In the short period since the launch of Santen Cell Therapy, we have made significant progress in forging relationships with key stakeholders in the field. Earlier this year, we became members of three influential organizations in the regenerative medicine and rare disease fields: The Alliance for Regenerative Medicine (ARM), the International Society for Cell Therapy (ISCT) and the European Organization for Rare Diseases (EURORDIS).

Through these memberships, not only do we aim to grow our understanding of the sector and keep up with latest developments, but we can also contribute to shaping the ecosystem and develop direct relationships with key stakeholders in the field.

BPR: What are the pressing issues in both the ophthalmology and advanced therapy spaces?

Santen:​ The recent World Health Assembly global targets on eye health and the UN General Assembly Resolution on Vision for Everyone recognize the importance of vision for considerations including employment, education and mental health. In developed countries, basic access to eye care services is already widely available. Advanced therapies offer the potential to address currently unmet medical needs and could significantly improve quality of life for patients with rare eye diseases.

The ongoing revision of key legislative files at the EU level, for example, should be leveraged in order to develop an ecosystem with fewer hurdles for patients to access advanced therapies. The upcoming review of the EU Directive on Cross-border Healthcare will be critical in simplifying the approval process for accessing treatments abroad and reducing timelines for patients seeking potentially life-changing therapies. Currently, patients often have to pay upfront for the cost of their treatment abroad and request reimbursement later. One solution to this would be to enable direct billing between health institutions.

Santen aims to work with key organizations and stakeholders in the field in order to find solutions to address such pressing issues.

BPR: What are Santen’s short and long-term goals in this space?

Santen: ​The Santen Cell Therapy organization has been set up to develop advanced therapies that address unmet needs in orphan ophthalmic diseases, with a current focus on accelerating the retinitis pigmentosa program for hRPC therapy. That therapy holds a regenerative medicine advanced therapy designation in the US and an orphan drug status in the US and the EU.

Phase 2b clinical trials have been completed and we are currently in the process of planning the phase 3 trials.

In the long term, we aim to build a sustainable platform based on a business model that combines the agility of a startup with Santen’s expertise and heritage, in order to develop innovations at a pace that can benefit society.

Given that we are operating in a new field evolving at an unprecedented rate, our strategy must remain highly agile and innovative. In clinical development, for example, diagnostic methods and outcome measures are still under discussion, leading to many unknowns.

In patient advocacy, the distinctive characteristics of the rare disease business model and the lack of existing relationships with key stakeholders in the field present certain challenges. However, we see this as an opportunity for Santen to establish itself as a leader within the rare disease space in ophthalmology and become a trusted partner for patients suffering from inherited retinal diseases.  

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