Clinical Trials
55
0 active
Approvals
29
Total approvals
Agencies
4
Regulatory bodies
Founded
N/A
Completed
53
96.4%
Recruiting
2
3.6%
- Shield Therapeutics posted H1 2026 total revenues of $30.4 million, up from $21.5 million in H1 2025, driven by a $7.9 million milestone payment from ASK in China and 21% prescription growth. - ACCRUFeR® (ferric maltol) achieved c.102,000 prescriptions dispensed in H1 2026, and the company signed its first Group Purchasing Organisation contract providing access to over 400 clinics. - MEDLEAP Pharma enrolled the first patient in a Phase II clinical trial of ferric maltol for pulmonary arterial hypertension in Japan, with a planned Phase III trial to follow. - Pediatric indication extensions were secured in the US (ages 10+), Europe (ages 12+), and the UK (ages 12+), expanding the addressable patient population.
- Swissmedic has approved PEDMARQSI (sodium thiosulfate solution) as the first and only treatment authorized in Switzerland for preventing cisplatin-induced hearing loss in pediatric cancer patients aged 1 month to 18 years. - Clinical trials demonstrated approximately 50% reduction in cisplatin-induced ototoxicity when sodium thiosulfate was administered alongside cisplatin compared to cisplatin alone, while maintaining chemotherapy efficacy. - The approval addresses a significant unmet medical need, as cisplatin-induced hearing loss can begin with the first dose and have lifelong impacts on children's speech, language development, and quality of life. - PEDMARQSI previously received European Medicines Agency approval in May 2023 and UK authorization, reflecting successful navigation of diverse regulatory pathways for rare pediatric medicines.
- The European Medicines Agency's CHMP issued a positive opinion recommending approval of mavorixafor (XOLREMDI®) for WHIM syndrome treatment in patients aged 12 years and older. - The recommendation is based on results from the pivotal Phase 3 4WHIM trial, which demonstrated significant improvements in neutrophil and lymphocyte counts and a 40% reduction in infection scores. - Final approval decision from the European Commission is expected in Q2 2026, potentially making this the first authorized treatment for WHIM syndrome in Europe. - Norgine will commercialize the therapy across Europe, Australia and New Zealand following regulatory approval under a licensing agreement with X4 Pharmaceuticals.
- Vir Biotechnology granted Norgine exclusive commercial rights to tobevibart and elebsiran combination therapy for chronic hepatitis delta in Europe, Australia, and New Zealand for €55 million upfront and up to €495 million in milestones. - The combination therapy achieved 66% hepatitis delta virus RNA target not detected in Phase 2 SOLSTICE trial, representing a potentially first-of-its-kind treatment for the most severe form of chronic viral hepatitis. - Norgine will contribute 25% of ongoing ECLIPSE registrational program costs, while Vir retains commercialization rights in the United States and extends cash runway into Q4 2027.
- The Australian Therapeutic Goods Administration has approved IFINWIL® (eflornithine) for treating adults and children with high-risk neuroblastoma who have responded to prior multimodality therapy. - High-risk neuroblastoma predominantly affects young children with an average survival rate of only 50%, and approximately 25 Australian children are diagnosed with this aggressive form annually. - The approval comes through Project Orbis, an FDA initiative facilitating collaborative international regulatory reviews to expedite patient access to innovative cancer therapies worldwide.
- Norgine has entered a definitive agreement to acquire Theravia from Mérieux Equity Partners, adding key rare disease treatments SIKLOS® for sickle cell disease and ORPHACOL® for genetic bile disorders to its portfolio. - The acquisition aligns with Norgine's growth strategy, creating a European-based rare disease champion with six core products that will serve as key growth drivers in the medium-to-long term. - This transaction follows Norgine's recent deals with Fennec Pharma for PEDMARQSI® and X4 Pharma for mavorixafor, establishing the company as a partner of choice for commercializing rare and specialty pharmaceuticals in Europe.
- X4 Pharmaceuticals' Marketing Authorization Application (MAA) for mavorixafor to treat WHIM syndrome is under EMA review, potentially the first EU-approved drug for the condition. - Mavorixafor's MAA is supported by Phase 3 trial results, showing reduced infection rates and severity in WHIM syndrome patients with a good safety profile. - Norgine and X4 Pharmaceuticals have an exclusive licensing agreement to commercialize mavorixafor in Europe, Australia, and New Zealand upon regulatory approval. - Mavorixafor, already approved in the U.S. as XOLREMDI, is a CXCR4 antagonist, aiming to increase circulating neutrophils and lymphocytes in WHIM syndrome patients.
• Norgine has submitted a marketing authorization application to the EMA for eflornithine to treat high-risk neuroblastoma (HRNB). • The application follows prior submissions in Australia, Switzerland, and the United Kingdom under Project Orbis. • Eflornithine is intended as an oral maintenance therapy to reduce relapse risk in pediatric and adult HRNB patients. • FDA approved eflornithine in December 2023 based on trials showing improved event-free and overall survival.
- Shield Therapeutics' FORTIS/ST10-01-305 trial met all primary endpoints, demonstrating a clinically relevant increase in hemoglobin (Hb) levels over 12 weeks in children with iron deficiency. - In children aged 2-17, the mean Hb increase was 1.25 g/dL, while infants showed an increase of 1.77 g/dL, compared to 1.15 g/dL in the ferrous sulphate group. - No patients in the ferric maltol group discontinued the study due to treatment-related adverse events, highlighting the safety and tolerability of the oral liquid suspension. - Shield Therapeutics plans to submit regulatory applications to the FDA and EMA in the first half of 2025 for a pediatric indication, with potential for a €1m milestone payment from Norgine.
- Norgine B.V. has submitted marketing authorization applications for eflornithine in Australia, Switzerland, and the United Kingdom for high-risk neuroblastoma (HRNB). - The submissions are part of Project Orbis, an initiative to expedite the approval of innovative oncology products across multiple countries. - Eflornithine is intended as an oral maintenance therapy to reduce the risk of relapse in pediatric and adult patients with HRNB. - FDA approved eflornithine in December 2023 based on trials showing improved event-free and overall survival compared to standard of care.
