The U.S. FDA has approved Dawnzera (donidalorsen) from Ionis Pharmaceuticals Inc. as a prophylactic therapy for rare and genetic disease hereditary angioedema (HAE). The approval came as expected, as the NDA had a PDUFA date of Aug. 21. The ligand-conjugated antisense targeting the prekallikrein mRNA is designed to prevent HAE attacks. Swelling is a dominant symptom, including the neck and hands, and it affects about 7,000 people in the U.S. About 25% of those diagnosed with HAE do not have a family history of the disease. Kyle Jenne, Ionis chief global product strategy office, said on a call soon after the approval was announced, that the cost will start at $740,000 annually. Dawnzera comes with an autoinjector for dosing every eight weeks and takes about 10 seconds to self-administer. 

Nicox’s NCX-470 noninferior to latanoprost in second phase III

Showing similar results as the first phase III trial completed in 2022, Nicox SA’s nitric oxide-donating bimatoprost eye drop, NCX-470, met the primary endpoint in the phase III Denali trial for open-angle glaucoma or ocular hypertension, clearing the way for regulatory filings in both the U.S. and China. Although the once-daily dosing of NCX-470 0.1% showed noninferiority in lowering intraocular pressure (IOP) from baseline compared to most commonly used latanoprost 0.005% – which entered the market in 1996 and is now available in generic form – Nicox’s eye drop failed to achieve statistical superiority on a secondary analysis of time-matched change from baseline IOP, showing a difference of up to 0.8 mmHg in favor of NCX-470. The phase III Mont Blanc study revealed similar findings three years ago. Still, the consistent results support submissions to regulators, according to Nicox. 

Researchers find new pain gene and analgesic drug target

An investigation of the epidemiology and clinical characteristics of neuropathic pain in the UK Biobank has led to the discovery of a new pain gene and potential analgesic drug target in the peripheral nervous system. The gene, SLC45A4, codes for a transporter which is involved in trafficking polyamines that are known to be involved in pain across the cell membrane. Subsequent detective work confirmed how the protein operates at a molecular level, investigated its expression in pain pathways in mouse models and elucidated the 3D structure. The findings lay the ground for drug discovery, said David Bennett, head of the Division of Clinical Neurology at Oxford University, who is senior author of a paper in Nature Aug. 21, 2025, describing the research. “We will try and see if we can target SLC45A4 as a means to provide analgesia ….. We will be using our knowledge of the structure to do this,” he told BioWorld. 

EU, US lock in 15% max Rx tariff

No matter what level of tariff the Trump administration ends up imposing on biopharmaceuticals across the world, drugs and their precursors originating in the EU will face no more than a 15% tariff when imported to the U.S., according to the joint statement the EU and U.S. issued today regarding their trade framework agreement. While that’s better than the 25% minimum U.S. President Donald Trump has teased as a starting point for a global biopharma sector tariff, it’s still a 15-fold increase over the 0% tariff the U.S. has maintained on medicines since at least the 1990s. In addition to the tariffs, the agreement lays out a cooperative strategy for the trade partners to defend against export restrictions and other harmful practices by third countries that impact the life sciences and other industry sectors.

South Korea forms new organoid consortium for nonanimal testing 

South Korea’s Ministry of Food and Drug Safety (MFDS) launched a new industry-academia-research consortium to support the nation’s organoid industry, expected to grow in light of the U.S. FDA’s shift away from animal testing in the development of novel drugs. “We hope that the consortium launched today will serve as a communication hub where the industry, academia and research institutions can work closely together and through which domestic technology can advance globally and lead the standardization [of organoid products] worldwide,” MFDS Minister Oh Yu-kyoung said.

Generative AI comes up with novel antibiotic structures

Researchers at the Massachusetts Institute of Technology have developed a generative AI model that was able to generate novel antibiotic structures from either chemical fragments or de novo, starting from ammonia, methane, water or no starting point at all. “The big accomplishment here is to show that generative AI can be harnessed in drug discovery, in our case antibiotics, really to tap into untapped chemical spaces quite efficiently and in a directed manner, and so based on modest training data, one can make pretty big strides towards coming up with molecules with new properties,” senior author Jim Collins told BioWorld. 

Abcellera soars 121%, leads biopharma gainers on analyst buzz

Over June and July, 13 biopharma stocks in the BioWorld Biopharmaceutical Index (BBI) posted gains, while nine declined. Topping the list of gainers was Abcellera Biologics Inc., which climbed 121% over the two-month span. On July 7, Leerink Partners resumed coverage, with analysts Faisal A. Khurshid and Heidi Jacobson noting they “like the story of a legacy antibody developer transitioning into a clinical-stage biotech.”

MedPAC anticipates doubling of Medicare spending by 2032

The Medicare Payment Advisory Commission (MedPAC) said in a recent report that Medicare spending in the U.S. is poised to nearly double to $1.9 trillion over the coming eight years, a figure that is likely to apply ever-increasing price pressures on makers of devices, diagnostics and pharmaceuticals.

Also in the news

Aligos, Alvotech, Alzecure, Anavex Life Sciences, Astellas, Belhaven Biopharma, Biogene, Calidi Biotherapeutics, Celldex, Cytokinetics, Dong-A ST, Madrigal, Mural Oncology, Novabay, Redhill Biopharma, Rezolute, Rocket Pharmaceuticals, Saniona, Sensory Cloud, Tevogen Bio, Tharimmune, XRA 5, Xspray Pharma, Xtalpi