Notizia - Recent Pharma, Healthcare and Biotech Happenings
Apr 01, 2025
Table of Contents
The FDA has approved Qfitlia (fitusiran), the first antithrombin-lowering therapy for routine prophylaxis to prevent or reduce bleeding episodes in hemophilia A or hemophilia B patients (ages 12+) with or without inhibitors. Approval is based on ATLAS Phase III trials, which showed a 71% reduction in annualized bleeding rates (ABR) for patients without inhibitors and a 73% reduction for those with inhibitors. Qfitlia uses small-interfering RNA technology, allowing low-frequency, subcutaneous dosing.
“Current treatment options force trade-offs between bleed control and convenience,” said Phil Gattone, President and CEO, National Bleeding Disorders Foundation. “Qfitlia offers a novel approach, providing effective bleed protection while reducing dosing frequency.”
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By lowering antithrombin (AT)—a protein that inhibits clotting—Qfitlia increases thrombin generation to restore hemostasis. It offers the fewest doses of any prophylactic hemophilia therapy, with as few as six injections per year. The FDA also cleared Siemens Healthineers’ INNOVANCE Antithrombin assay as a companion diagnostic to measure AT levels in patients prescribed Qfitlia. Sanofi is launching HemAssist, a support program for patients, covering financial assistance, insurance support, and education.
“This approval underscores our commitment to innovation in rare blood disorders,” said Brian Foard, Executive VP, Head of Specialty Care, Sanofi. “Qfitlia can reshape the hemophilia landscape with its effective bleed protection, infrequent dosing, and simplified administration.”
Qfitlia comes with potential risks, including thrombotic events, gallbladder disease, and hepatotoxicity. The FDA granted Orphan Drug, Fast Track, and Breakthrough Therapy designations for hemophilia treatment. Regulatory submissions are under review in Brazil and China, with a decision in China expected in late 2025.
The FDA has approved AstraZeneca’s IMFINZI (durvalumab) in combination with chemotherapy as the first perioperative immunotherapy for muscle-invasive bladder cancer (MIBC). The approval, granted after Priority Review, is based on NIAGARA Phase III trial results, which showed a 32% reduction in recurrence risk and a 25% reduction in death risk compared to neoadjuvant chemotherapy alone. The trial, presented at ESMO 2024 and published in The New England Journal of Medicine, highlighted the urgent need for better treatment options, as nearly half of MIBC patients experience disease recurrence despite standard chemotherapy and surgery.
“This approval is a major breakthrough for MIBC patients, significantly extending lives in the NIAGARA trial,” said Dr. Matthew Galsky of Mount Sinai. “The regimen has the potential to transform care.” Dave Fredrickson, AstraZeneca’s EVP of Oncology, added, “IMFINZI represents a paradigm shift, bringing the first perioperative immunotherapy to MIBC patients and addressing a significant unmet need.”
The NIAGARA trial demonstrated that 67.8% of patients treated with the IMFINZI regimen were event-free at two years, compared to 59.8% in the comparator group. Additionally, 82.2% of patients receiving IMFINZI were alive at two years, versus 75.2% in the control arm. The treatment was generally well tolerated, with no new safety concerns. In February 2025, the NCCN Clinical Guidelines added IMFINZI’s perioperative regimen as a Category 1 Recommended treatment for MIBC.
“There is a critical need for better treatments in MIBC,” said Meri-Margaret Deoudes, CEO of the Bladder Cancer Advocacy Network. “The approval of this regimen is a game-changer for patients and their families.”
IMFINZI is also approved in Brazil for this setting, with regulatory reviews ongoing in the EU, Japan, and other countries.
Beam Therapeutics announced that the FDA has cleared the IND application for BEAM-302, a precision genetic medicine for alpha-1 antitrypsin deficiency (AATD). BEAM-302, a liver-targeting base editing therapy, aims to correct the PiZ mutation, which causes AATD-related lung and liver disease. There are currently no approved curative treatments for AATD.
“This clearance is a critical step toward delivering a potential breakthrough for AATD patients in the U.S.,” said Giuseppe Ciaramella, Ph.D., President of Beam. “Our initial Phase I/II data demonstrated the first-ever clinical genetic correction of a disease-causing mutation, and we are focused on advancing enrollment and expanding trial sites.”
BEAM-302 is currently being tested in a Phase I/II clinical trial assessing its safety, tolerability, pharmacokinetics, and efficacy. The trial consists of Part A (AATD patients with lung disease) and Part B (patients with mild to moderate liver disease, with or without lung disease). The therapy has also received clinical trial authorization in the UK, Australia, New Zealand, the Netherlands, and Ireland.
Beam plans to continue dose escalation in Part A and expects to present updated data in late 2025. The company will also initiate dosing in Part B later this year, marking a major step toward establishing BEAM-302 as a one-time genetic treatment for AATD.
AstraZeneca’s CALQUENCE (acalabrutinib) plus bendamustine and rituximab has been recommended for EU approval for previously untreated mantle cell lymphoma (MCL) in adults ineligible for stem cell transplantation. The CHMP’s positive opinion is based on the ECHO Phase III trial, which showed a 27% reduction in disease progression or death vs. chemoimmunotherapy alone.
“The ECHO trial demonstrated the significant benefits of the CALQUENCE combination in managing this rare and aggressive cancer,” said Dr. Martin Dreyling of University Hospital LMU Munich. “This recommendation is a major step for older MCL patients needing both efficacy and tolerability.”
CALQUENCE plus bendamustine and rituximab has already been approved in the US and other countries, with regulatory reviews ongoing in Japan and several other regions. The CHMP also recently recommended CALQUENCE monotherapy for relapsed or refractory MCL, reinforcing its potential to reshape the standard of care.
“If approved, CALQUENCE would be the first BTK inhibitor for 1L MCL in Europe, offering patients a significant PFS advantage,” said Susan Galbraith, EVP, Oncology R&D at AstraZeneca. “This marks a critical advancement in treatment options.”
Ionis Pharmaceuticals and AstraZeneca have received European Union (EU) approval for WAINZUA (eplontersen), a once-monthly RNA-targeted therapy for hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adult patients with stage 1 or stage 2 polyneuropathy. This makes WAINZUA the only approved ATTRv-PN treatment in the EU that can be self-administered via an auto-injector.
The European Commission’s (EC) decision, based on a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), was supported by data from the NEURO-TTRansform Phase III trial. Over 66 weeks, WAINZUA demonstrated sustained reduction in serum transthyretin (TTR) concentration, significant improvement in neuropathy impairment (measured by mNIS+7), and better quality of life outcomes compared to an external placebo. The therapy also showed a favorable safety and tolerability profile throughout the trial.
Brett P. Monia, Ph.D., CEO of Ionis Pharmaceuticals, emphasized the impact of the approval, stating:
“Today’s approval of WAINZUA in Europe offers adults with ATTRv-PN a new, self-administered treatment option that provides consistent suppression of transthyretin production and improves neuropathy impairment and quality of life. With approvals in North America, the UK, and now across the EU, we are proud of the continued progress as we and our partner, AstraZeneca, rapidly and effectively deliver WAINZUA to people around the world.”
ATTRv-PN is a debilitating genetic disorder that progressively damages peripheral nerves, leading to motor disability within five years and often proving fatal within a decade if left untreated. Current treatment options remain limited, making WAINZUA a significant advancement in disease management. WAINZUA aims to slow disease progression and improve patient outcomes by reducing TTR production at its source.
Already approved in the U.S. (marketed as WAINUA), Canada, and the UK, WAINZUA continues to expand globally. As part of a joint development and commercialization agreement, AstraZeneca and Ionis are working on securing further regulatory approvals worldwide, with AstraZeneca holding exclusive commercialization and development rights outside the U.S.
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