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The accelerated approval for the tablet is based on the reduction of alkaline phosphatase levels. Credit: LightField Studios/ Shutterstock. Ipsen has received US Food and Drug Administration (FDA) accelerated approval for Iqirvo (elafibranor) 80mg tablets for primary biliary cholangitis (PBC). Recommended Buyer's Guides Buyer's Guide Leading Guide to Compliance Software for the Pharmaceutical Industry Buyer's Guide Leading Guide to Clinical Packaging Companies in contract Manufacturing for the Pharmaceutical Industry The treatment is indicated for usage in combination with ursodeoxycholic acid (UDCA) for adult patients with an inadequate response to UDCA, or as monotherapy in patients who are intolerant to UDCA. Ipsen will handle the marketing and commercialisation of elafibranor under the trademark name Iqirvo and will make it available immediately for prescription in the US for eligible patients. The accelerated approval is based on the reduction of alkaline phosphatase levels, although improvements in survival or liver decompensation event prevention are not established. In the Phase III ELATIVE trial, 13 times more patients attained the composite primary endpoint of biochemical response following Iqirvo plus UDCA treatment than did so with placebo plus UDCA. See Also: NPX-267 by NextPoint Therapeutics for Triple-Negative Breast Cancer (TNBC): Likelihood of Approval PF-07960613 by Pfizer for Respiratory Syncytial Virus (RSV) Infections: Likelihood of Approval Secondary endpoints showed ALP level normalisation in only Iqirvo-treated patients. Continued approval for this indication will depend on results and clinical benefits observed in confirmatory trials. Iqirvo is not recommended for individuals with decompensated cirrhosis, characterised by conditions such as ascites, variceal bleeding or hepatic encephalopathy. GENFIT discovered and developed elafibranor with Ipsen licensing the sole global rights for the asset in 2021, excluding China, Hong Kong, Macau and Taiwan. GENFIT is entitled to receive milestone payments of €89m ($95.7m) from Ipsen. This comprises €48.7m on the first commercial sale of Iqirvo in the US, in addition to a €13.3m in milestone payment obtained in February 2024. Ipsen Research and Development head and executive vice-president Christelle Huguet stated: “For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC. Left unmanaged, PBC can progress, leading to liver failure and in some cases, the need for a liver transplant. “Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone. Iqirvo is therefore a much-needed treatment option and the first new medicine for PBC in nearly a decade.” The latest development comes after Ipsen expanded its ongoing oncology research partnership with Marengo Therapeutics to advance T cell engagers using TriSTAR, Marengo’s antibody fusion platform.

The approval of ganaxolone is based on results from a Phase III clinical trial in subjects aged two to 19 years. Credit: LightField Studios / Shutterstock.com. The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Marinus Pharmaceuticals Emerald’s ganaxolone, marketed as Ztalmy, an anti-seizure medication for cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder (CDD). CDD is a rare genetic ailment characterised by early-onset seizures and severe developmental delays due to mutations in the CDLK5 gene, essential for normal brain development. Ganaxolone, administered orally via a dosing syringe, is a steroid that targets specific brain receptors to prevent epileptic seizures. The approval of ganaxolone is based on results from a Phase III clinical trial. The double-blind placebo-controlled study involved 101 subjects aged two to 19 years. See Also: BeiGene secures accelerated FDA approval for follicular lymphoma treatment FDA approves BMS’ Opdivo for urothelial carcinoma The subjects were randomised with 50 receiving ganaxolone and 51 a placebo over 17 weeks. Those treated with ganaxolone showed a significant reduction in 28-day seizure frequency, averaging almost 30%, versus a 6.5% reduction for those on the placebo at 13 weeks. Side effects linked to ganaxolone are drowsiness or sleepiness and fever. The MHRA will monitor the safety and efficacy of the drug. Individuals experiencing adverse effects are encouraged to consult their healthcare provider and report the incident through the MHRA Yellow Card scheme, accessible online or via mobile app stores. MHRA Healthcare Quality and Access interim executive director Julian Beach stated: “Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us. “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. “As with all medicines, we will keep its safety under close review.” The latest development comes after the MHRA granted marketing authorisation for a new formulation of Amgen ’s XGEVA (denosumab) to prevent serious bone-related complications.

Lymphir is an IL-2-based immunotherapy for the treatment of relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. Credit: Shutterstock/LightField Studios Citius Pharmaceuticals is eyeing an approval decision and a potential commercial launch for its lead lymphoma therapy Lymphir by September or October 2024, said the company’s CEO Leonard Mazur in an In an exclusive interview with Pharmaceutical Technology. On 13 February, Citius announced that it had resubmitted its biologics license application (BLA) for Lymphir (denileukin diftitox/E7777) to the US Food and Drug Administration (FDA). The company expects the FDA to issue and package a new Prescription Drug User Fee Act (PDUFA) date within thirty days of the resubmission. Mazur predicts that the new PDUFA date will be set for a six-month timeframe. Lymphir is an IL-2-based immunotherapy for the treatment of relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. The drug binds to IL-2 receptors on the cell surface, leading to the inhibition of protein synthesis and subsequently cell death. In 2021, Citius acquired a license to develop Lymphir from Dr Reddy’s Laboratories , completing a Phase III trial for the drug in December 2021. In 2022, the company filed a BLA for the drug, but received an FDA complete response letter in July 2023. “We knew there was a small deficiency in that filing, but we felt that we would still get it approved,” says Mazur. He explains that the rejection was based on a manufacturing test on the final product that had been passed but had not yet been validated by the FDA, and the company has now resolved this issue. See Also: Jubilant Pharmova files patent for process for preparing highly concentrated allergenic intermediate from dilute extract Dexamethasone sodium phosphate by Scilex Holding for Back Pain: Likelihood of Approval Citius has transferred Lymphir over to a subsidiary company, Citius Oncology, with plans to have the subsidiary acquired through a special purpose acquisition company (SPAC). “This will give us a Nasdaq listing… Once the SPAC acquires the subsidiary, it will be renamed and will start to trade separately on NASDAQ at around May,” says Mazur. He explains that this approach should prevent the dilution of shareholders in Citius Pharmaceutical and allow the company to separately raise finances for the launch of Lymphir. Citius is also collaborating with Eversana for its post-marketing operations such as warehousing, shipping and billing of the drug. The New Jersey-based company also completed enrolment for a Phase III trial (NCT02901717) for its antibody lock therapy Mino-lok (edetate disodium + ethyl alcohol + minocycline hydrochloride), as per an announcement on 2 January. Citius is developing this therapy for the treatment of catheter-related blood stream infections. Mazur now forecasts a topline data readout for the trial in May or June 2024, and further plans will be made following the data readout.