Quotient Sciences Extends Partnership with Ipsen for Manufacture of Ultra-Rare Disease Treatment

Quotient Sciences, a contract research, development, and manufacturing organization, announced on February 26, 2026 that it is extending its commercial supply partnership with biopharmaceutical company, Ipsen.1 Under the partnership, Quotient will manufacture Sohonos (palovarotene), an FDA-approved, highly potent molecule for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease that impacts fewer than 1000 people worldwide. The partnership will ensure that a supply of the FOP treatment is secure.

Because of its highly potent nature, palovarotene requires specialized handling and containment. Ipsen has invested in new equipment at Quotient’s Boothwyn, PA facility for the manufacture of the product. This investment includes a pneumatic closed transfer system, for safe material transfer without impacting blend segregation, and a flexible dispensing isolator to enhance operator safety and remove special cleaning requirements.

“This significant investment in advanced containment and manufacturing technology at our Boothwyn facility underscores Quotient Sciences’ unwavering commitment to supporting patients with ultra-rare diseases,”Marlene Leuenberger, VP and General Manager, Quotient Sciences, Philadelphia, said in a press release.1 “By expanding our capabilities in handling highly potent compounds, we are not only ensuring a reliable supply of critical therapies like Sohonos (Palovarotene) for the FOP community, but also reinforcing our promise to deliver flexible, high-quality solutions for our partners with specialized needs especially for highly potent compounds.”

What other targeted medicines is Ipsen developing?

In Europe, Ipsen’s monotherapy, Ojemda (tovorafenib), was given a positive opinion by the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) for conditional marketing authorization.2 Ojemda (tovorafenib) is a treatment for pediatric low-grade glioma (pLGG) with harboring a BRAF fusion or rearrangement or BRAF V600 mutation, who have progressed after one or more prior systemic therapies, in patients 6 months of age and older. According to the company, BRAF altered pLGG causes problems with sight, speech, and neurological problems in children.

“For children diagnosed with pediatric low‑grade glioma, the journey is long and complex, with limited treatment options and no clear standard of care,” said Christelle Huguet, PhD, EVP and head of Research & Development, Ipsen, in a press release.2 “Innovating in pediatric oncology is challenging, and genuine breakthroughs are rare. The evidence supporting tovorafenib, which acts on tumor growth driven by an abnormal BRAF gene, is an important development. The positive CHMP opinion moves us closer to delivering a meaningful targeted therapy for these patients and their families.”

“Treatment options for relapsed/refractory pediatric low-grade glioma have remained limited for decades, which is why a targeted therapy like tovorafenib has the potential to transform how we manage this disease in particular the most common subtype carrying the BRAF-Fusion oncogene,” said Professor Olaf Witt, director, Translational Pediatric Oncology, Hopp Children’s Cancer Center Heidelberg, in the press release.2 “As a treating physician, I am encouraged by the prospect of introducing a new treatment option for this challenging condition in Europe.”

The CHMP opinion was based on data from a pivotal phase 2 FIREFLY-1 study. Tovorafenib was evaluated in 137 children and young adults with relapsed or refractory BRAF-altered pLGG who had received at least one prior systemic therapy. The study showed there as an overall response rate of 71%, and the median time to response was 5.4 months with an 18-month median duration of response. Tovorafenib was well tolerated, according to the study, with grade 1 or 2 treatment-related adverse events.

References

1. Quotient Sciences Extends Commercial Manufacturing Partnership for Ultra-Rare Disease Treatment. Press Release. Quotient Sciences. February 26, 2026. https://www.quotientsciences.com/news/quotient-sciences-extends-commercial-manufacturing-partnership-ultra-rare-disease-treatment
2. Ipsen receives positive CHMP opinion for Ojemda® for the treatment as monotherapy of children with relapsed or refractory BRAF-altered pediatric low-grade glioma. Press Release. Ipsen. February 27, 2026. https://www.ipsen.com/press-release/ipsen-receives-positive-chmp-opinion-for-ojemda-for-the-treatment-as-monotherapy-of-children-with-relapsed-or-refractory-braf-altered-pediatric-low-grade-glioma-3246394/