Johnson & Johnson's Guselkumab Receives FDA Approval for Ulcerative Colitis; Roche Submits Risdiplam Tablets for SMA in China; Sanofi In-Licenses Novel Radiopharmaceutical AlphaMedix

Sep 13, 2024 20:17 CST Updated 20:17

Johnson & Johnson

Medical Device R&D and Manufacturer

Johnson & Johnson's Guselkumab Receives FDA Approval for Ulcerative Colitis Treatment

On September 11, Johnson & Johnson announced that the U.S. FDA had approved Tremfya (guselkumab) for the treatment of adult patients with moderately to severely active ulcerative colitis (UC).The company claims that Tremfya is the first and only dual-action inhibitor targeting interleukin-23 (IL-23) approved for the treatment of active ulcerative colitis, further solidifying Johnson & Johnson's leadership position in the field of inflammatory bowel disease.

Guselkumab is the world's first approved interleukin-23 (IL-23) inhibitor, which selectively binds to the p19 subunit of IL-23, inhibiting its interaction with the IL-23 receptor, and has shown good efficacy in various autoimmune diseases; it can also bind to the receptor CD64 on cells producing IL-23.

In July 2017, Guselkumab was first approved for marketing in the United States for the treatment of moderate to severe plaque psoriasis. Two years later, Guselkumab entered the Chinese market.

Currently, Guselkumab has been approved for the treatment of plaque psoriasis, active psoriatic arthritis, and UC. In addition, in June 2024, Johnson & Johnson submitted an sBLA to the FDA for the treatment of adult patients with moderate to severe active Crohn's disease (CD). The approval of this indication for UC was primarily based on data from the Phase 2b/3 QUASAR study.

Roche's SMA Oral Therapy Risdiplam Tablets Submitted for Marketing Approval in China

On September 12, the CDE website announced that the listing application for risdiplam tablets submitted by Roche has been accepted, with the specific indications yet to be disclosed.Public information shows that risdiplam, an innovative neuro-rare disease drug from Roche, is used to treat spinal muscular atrophy (SMA).The oral solution of this drug has been approved for marketing in China, and the dosage form of this marketing application is tablets.

SMA is one of the autosomal recessive genetic diseases causing infant mortality, resulting from mutations in the survival motor neuron 1 (SMN1) gene leading to defective SMN protein function.Risdiplam is an SMN2 gene mRNA splicing modifier.It modulates the splicing of SMN2 through dual-site specificity, promoting the retention of exon 7 and increasing the level of functional SMN protein.The drug can also penetrate the blood-brain barrier, distributing in both the central and peripheral systems, increasing SMN protein levels across multiple systemic areas, while maintaining stability.Risdiplam is currently or has already been evaluated in five global multicenter trials for patients with SMA.

In China, risdiplam was first approved by the Chinese NMPA for marketing in June 2021 for the treatment of SMA patients aged 2 months and above, ushering in a new era of oral SMA treatment. In June 2023, the drug received another NMPA approval, expanding its use to SMA patients aged 16 days and above. The previously approved product, risdiplam oral solution, is a liquid formulation that can be taken orally at home or administered through a feeding tube in liquid form.

Sanofi Introduces a Radiopharmaceutical

On September 12, Sanofi announced that it had entered into a licensing agreement with RadioMedix and Orano Med, a subsidiary of the Orano Group, to develop next-generation radioligand therapies.

RadioMedix and Orano Med's Collaboratively Developed Radiopharmaceutical AlphaMedix (212Pb-dotamtate) is a Key Focus for Sanofi. This drug is an α-particle emitting peptide conjugate targeting somatostatin receptors (SSTR). Currently, AlphaMedix has completed a Phase II study (ALPHAMEDIX 02) involving adult patients with gastroenteropancreatic neuroendocrine tumors (GEP-NET). In February this year, AlphaMedix received Breakthrough Therapy Designation from the FDA.

According to the licensing agreement, Sanofi will be responsible for the global commercialization of AlphaMedix, while Orano Med will handle the production of AlphaMedix. In return, RadioMedix and Orano Med will receive an upfront payment of 100 million euros (approximately 109 million US dollars) and milestone payments of up to 220 million euros (approximately 239 million US dollars), along with eligibility for tiered royalties.

Editor: Allen