Following a priority review, Apellis Pharmaceuticals Inc. received U.S. FDA approval of a supplemental NDA making pegcetacoplan the second marketed treatment for complement 3 glomerulopathy and the first for primary immune complex membranoproliferative glomerulonephritis. The approval to reduce proteinuria for those 12 and older with either of the two severe and rare kidney diseases came on the July 28 PDUFA date, a little more than four years after the company won approval of the targeted C3 therapy, branded Empaveli, for the rare blood disorder paroxysmal nocturnal hemoglobinuria. The average annual wholesale acquisition cost is currently set at $505,000 per patient, or $4,860 per vial. The most recent approval is based on phase III results from the Valiant study showing meaningful benefits at week 26 across key disease markers of proteinuria reductions, kidney function stabilization and the clearance of C3c staining.

PTC’s Sephience wins FDA nod for phenylketonuria

PTC Therapeutics Inc. will be launching its oral phenylketonuria (PKU) therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission. As with the Europe label, the FDA nod clears Sephience for broad use in adults and children living with PKU, including the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU. A natural precursor of enzymatic co-factor BH4, Sephience is indicated as a once-daily, oral therapy designed to reduce blood phenylalanine (Phe) levels.

Cellcentric, Draig help steady Q2 venture funding in UK

Total venture capital funding in U.K. biotech was £1.23 billion (US$1.64 billion) in the first half of 2025, almost matching the 2023 total of $1.66 billion and looking to be in position to equal the 2024 12-month total of $2.8 billion.

Novo claws back expectations and the stock slumps

With reduced sales and operating expectations for the rest of the year, Novo Nordisk A/S is on the receiving end of a huge stock drop. Lower U.S. sales of semaglutide blockbusters Wegovy and Ozempic for treating obesity and diabetes are at the heart of the plunge. In May, the Danish company had expected 13% to 21% sales growth but now is looking at only 8% to 14%. Operating profit expectations were lowered, too, now at 10% to 16% instead of May’s forecast of 16% to 25%. Getting the blame for Wegovy was “the persistent use of compounded GLP-1s” plus slower-than-expected market expansion and competition. For a big pharma, where the stock drops are usually more modest, the shares (NASDAQ:NVO) had fallen off a cliff at midday, trading 22% downward at $54 each.

Clarity raises AU$203M to advance copper-based radiopharma pipeline

Radiopharma Clarity Pharmaceuticals Ltd. raised AU$203 million (US$132.22 million) in an institutional placement on Australia’s Securities Exchange to advance its late-stage pipeline of copper-based radiopharmaceuticals. The placement consisted of 48.485 million new fully paid ordinary shares priced at AU$4.20 per share, representing a 2.2% premium to Clarity’s last closing price. Bell Potter Securities Ltd. acted as sole lead manager. As previously reported by BioWorld, Clarity raised AU$121 million in March 2024.

Sarepta to restart shipments of Elevidys in DMD

The U.S. FDA told Sarepta Therapeutics Inc. that the agency may lift the voluntary pause on shipments of Elevidys (delandistrogene moxeparvovec) for ambulatory patients with Duchenne muscular dystrophy (DMD), and the firm plans to resume sending out the drug again “imminently.” Last week, shipments were paused while the FDA examined safety information for the DMD gene therapy, which has been linked to several patient deaths. Shares of the company (NASDAQ:SRPT) were trading at $17.28, up $3.42, or 24%.

Also in the news

Aclaris, Acumen, Adaptimmune, Aim Immunotech, Akeso, Alfasigma, Alteogen, Alterity, Ascendis, Assembly, Astrazeneca, Bayer, Beone Medicines, Boehringer Ingelheim, Celcuity, Cervomed, Clarity, Deciphera, Ikena, Inmagene, Lantern, Lenz, Maat, Maia, Maplight, Nkgen, Phio, Phoremost, Promis, Realta, Recognify, Re-Vana, Sage, Servier, Shanton, Simris, South Rampart, Supernus, Taran, Theralase, Urogen, Zevra