IASO Bio's Equecabtagene Autoleucel Granted Orphan Drug Status by Saudi Authorities

IASO Biotherapeutics Achieves Major Milestone with Orphan Drug Designation

In a significant development for the biopharmaceutical sector, IASO Biotherapeutics, often referred to as IASO Bio, has announced that its innovative CAR-T cell therapy known as Equecabtagene Autoleucel (or FUCASO) has received Orphan Drug Designation (ODD) from the Saudi Food and Drug Authority (SFDA). This designation is aimed at bolstering the treatment landscape for patients suffering from relapsed or refractory multiple myeloma (R/R MM) who have exhausted standard treatment options.

What is Orphan Drug Designation?

Orphan Drug Designation is a regulatory status granted to drugs intended to treat rare diseases, typically those affecting fewer than 200,000 people in any given market. The SFDA's ODD is designed to encourage the development of effective therapies for these rare conditions by providing various incentives, including expedited review processes and development assistance. By achieving this designation, IASO Bio's Equecabtagene Autoleucel is now positioned to directly submit a New Drug Application (NDA) in Saudi Arabia, a move expected to streamline the approval timeline significantly, ultimately allowing for earlier access to this pioneering therapy for patients in need.

IASO Bio's Vision and Commitment

Jinhua Zhang, the Founder and CEO of IASO Biotherapeutics, expressed excitement about the recent approval, stating, "We are delighted that Equecabtagene Autoleucel has been granted the Orphan Drug Designation by the SFDA. This once again underscores international recognition of the clinical value of this groundbreaking therapy. Saudi Arabia not only marks an essential launch point for our expansion into the Middle East market but also aligns perfectly with our overarching strategy of 'Go Global.'"

The company is committed to collaborating with local regulatory bodies, medical institutions, and key partners to expedite the approval processes for equecabtagene autoleucel in the region, thereby delivering clinical benefits to patients battling multiple myeloma.

Understanding Multiple Myeloma

Multiple myeloma is recognized as the second most common type of blood cancer worldwide. Data from Globocan reflects that, as of 2022, the global incidence of this disease was approximately 1.8 per 100,000 individuals, with a 5-year prevalence rate of 6.8 per 100,000. Despite advancements in treatment methodologies, this hematologic malignancy remains primarily incurable, with patients frequently experiencing multiple relapses and developing resistance to several therapeutic agents. Consequently, there is a pressing demand for novel treatment options capable of yielding substantial and durable responses for patients with relapsed or refractory MM.

The Innovation Behind Equecabtagene Autoleucel

What sets Equecabtagene Autoleucel apart as a pioneering therapy is its design as a fully human anti-BCMA CAR-T cell treatment. This innovative product employs lentiviral vectors for the transfection of autologous T cells, leading to a robust treatment response. The CAR structure includes a fully human single-chain variable fragment (scFv), along with essential components such as the CD8α hinge, transmembrane domain, and co-stimulatory and activation domains (4-1BB and CD3ζ respectively).

Research supporting the efficacy of FUCASO indicates rapid and powerful treatment outcomes, with demonstrated long-term persistence after administration. These characteristics create a potential for achieving sustained and profound remissions for patients suffering from multiple myeloma, thereby offering continued care and protection.

IASO Bio's Broader Pipeline and Commitment to Innovation

Founded with a vision to transform how oncology and autoimmune conditions are treated, IASO Bio is on a mission focused on the discovery and creation of innovative cell therapies and biologics. With a diverse portfolio encompassing over ten novel products, including Equecabtagene Autoleucel—which received Biologics License Application (BLA) approval in China in June 2023 and FDA Investigational New Drug (IND) approval for the treatment of R/R MM in December 2022—their commitment to medical innovation is evident.

As IASO Bio continues to leverage a robust management team and integrated clinical capabilities, the company strives to deliver transformative, accessible therapies aimed at fulfilling unmet medical needs, both in China and globally.

For more information on IASO Bio’s revolutionary approaches to cell therapies and the latest on Equecabtagene Autoleucel, please visit IASO Bio's official site.