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Bexlutry has been approved in the US as a generic – or 'radioligand equivalent' (RLE) – of Novartis' gastroenteropancreatic neuroendocrine tumour (GEP-NET) therapy Lutathera.

Bexlutry is based on lutetium Lu 177 dotatate, the same active ingredient in Lutathera, and is the first RLE product to be approved by the FDA.

Like Lutathera, it can be used to treat somatostatin receptor-positive GEP-NETs, including foregut, midgut, and hindgut neuroendocrine tumours in adults.

GEP-NETs are a rare and complex type of cancer that forms from neuroendocrine cells of the gastrointestinal tract or the pancreas, and represent around two-thirds of all NETs. (Pharmaphorum)

The FDA approved Joenja (leniolisib) for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in children aged 4 to 11 years weighing at least 27 kg.

This is the first treatment approved for APDS, a rare primary immunodeficiency caused by variants in PIK3CD or PIK3R1, in this age group. (Healio)

Scholar Rock won FDA approval for its drug treating a rare muscle-wasting disease, after facing delays due to a troubled manufacturing factory.

The injectable drug Isembyld (apitegromab) was approved in the US for adults and children two years of age and older with spinal muscular atrophy already receiving a survival motor neuron 2 (SMN2)-targeted treatment.

Isembyld is Scholar Rock’s first commercially approved therapy and first muscle-targeted treatment in the US for spinal muscular atrophy.

The FDA’s go-ahead comes a month after the company said it was removing a troubled manufacturing site in Bloomington, IN, owned by Novo Nordisk, from Isembyld’s application. (Endpoints)

Novartis is boosting its capabilities for brain drug delivery by acquiring a platform technology that Sironax developed to bring drugs across the protective membrane.

The deal comes as scrutiny mounts on Novartis’s neuroscience pipeline following recent clinical trial setbacks.

Novartis has been looking at the Sironax technology for more than a year. Last summer, the Swiss pharmaceutical company secured an exclusive option to buy the platform.

That deal permitted Novartis to evaluate the technology during an option period, after which it could buy it.

Sironax announced Wednesday that Novartis will exercise that right by paying $125 million. (MedCity News)

Newly renamed Novo has joined forces with fellow Danish biotech Orbis Medicines in a multi-drug deal worth up to $1.4 billion.

The agreement focuses on the development of oral cardiometabolic drugs, particularly a class of treatments called macrocycles.

The partnership, announced today, is intended to produce orally bioavailable macrocycle drug candidates for cardiometabolic targets, though the companies did not elaborate on which ones.

Orbis’ AI-based platform is designed to accelerate discovery of orally available biologics via ring-shaped molecules.

The biotech joins dozens of others in the hunt for next-generation drugs for weight loss, riding the wave of GLP-1 pill success led by Novo’s Wegovy and main rival Eli Lilly’s Foundayo. (BioSpace)

Biotech startup Sling Therapeutics is taking an oral drug for thyroid eye disease into late-stage testing, emboldened by a $123 million Series C financing that it announced Wednesday morning.

A surge of research into new drugs for thyroid eye disease or TED, an immune condition characterized by eye bulging and swelling, has fueled the advancement of companies such as Sling, Ethyreal Bio and Khartis in recent months. Other drugmakers, like Immunovant and Argenx, have seen prospective treatments for the inflammatory eye condition fall short in testing.

Sling is trying to stand out with an oral drug that could become a more convenient alternative to Amgen’s Tepezza and Viridian Therapeutics’ Lumvoa.

Like both of those medicines, Sling’s linsitinib blocks a protein called IGF-1R that drives inflammation in TED. But Tepezza and Lumvoa are antibodies administered via an injection or intravenous infusion.

An oral drug like Sling’s, which is taken twice daily, could have an advantage there, said Ryan Zeidan, the biotech’s president and CEO. (Biopharma Dive)

There's widespread agreement among experts that for the US to maintain its competitive edge, it needs to streamline its drug development process.

The pilot is part of Operation TrialBlazer, the Trump administration's initiative to restore American leadership in clinical trials.

The FDA would review components of the application on a rolling basis, rather than waiting for a full submission to begin a review.

This rolling review will "creat[e] certainty and reduc[e] unnecessary delays," Karim Mikhail, director of the FDA Center for Biologics Evaluation and Research, told reporters.

The agency will select up to 10 pairings of drug sponsors and research institutions for the pilot.

After Lykos’ MDMA-assisted therapy for post-traumatic stress disorder failed to win FDA approval in 2024, the space stepped back from the limelight.

But now they’re back, with Compass Pathways knocking on the regulator’s door and Definium Therapeutics and other psychedelic drugmakers hoping to file soon.

Also, Definium’s LSD-based therapy has been meeting or exceeding high expectations in recent clinical readouts. In June, the drug elicited “profound” efficacy in a late-stage depression trial, then just last week delivered again with a solid late-stage win in anxiety.

As Health Secretary Robert F. Kennedy Jr. works to phase out selective serotonin reuptake inhibitors (SSRIs)—a current mainstay of mental health treatment—the time could be ripe for the entrance of psychedelics onto the U.S. market.

The next 12 months are certain to be pivotal for the space.