In a win for the rare disease space, the U.S. FDA granted accelerated approval for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel) as the first gene therapy option for treating severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare genetic immune disorder characterized by an immunodeficiency predisposing those affected to recurrent and fatal infections. The approval, coming just ahead of the March 28 PDUFA date, clears Kresladi for use for pediatric patients with severe LAD-I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant

EMA to withdraw approval for Siga’s tecovirimat in treating mpox infections

As expected, the EMA is recommending withdrawal of the mpox indication for Siga Technologies Inc.’s tecovirimat, whilst maintaining its approval as a treatment for smallpox, cowpox and adverse reactions to vaccinia vaccines. The decision was reached after the EMA’s Committee for Medicinal Products for Human Use (CHMP) reviewed the label for the antiviral drug following its use in patients during the recent mpox outbreaks. On a more positive note, almost three years to the day since the FDA gave it the nod, Pharming Group NV’s Joenja (leniolisib), is being recommended for approval by EMA.

Novartis buying allergy firm Excellergy for $2B

Just five months after raising a $70 million series A round, newly launched Excellergy Inc. agreed to a $2 billion M&A transaction that would bring its trifunctional allergic effector cell response inhibitors (ECRIs) to Novartis AG. The company’s ECRIs disrupt the immunoglobulin E (IgE) and Fc epsilon RI (FcεRI) complex. They are able to remove IgE bound to FcεRI on the surface of mast cells and basophils without triggering activation. By intercepting and neutralizing circulating IgE, and by down-regulating the FcεRI complex, they can bring allergic reactions back to baseline levels. The company’s lead candidate, Exl-111, recently entered a phase I trial. The deal with Novartis validates the “transformative potential” of Excellergy’s ECRIs and brings it under the umbrella of the pharma’s development and commercialization expertise, said Todd Zavodnick, Excellergy’s CEO.

Xcath sees Iris robotic platform transforming stroke treatment

As robotic-assisted surgery platforms increasingly demonstrate that they can perform telesurgery safely, the question now is how soon will remote operations become part of routine clinical care. Eduardo Fonseca, CEO of Xcath Robotics Inc. reckons that within a decade the company’s Iris Surgical Robotic System could be in widespread use. Xcath recently demonstrated the telerobotic capability of the device, which enabled a neurosurgeon to successfully treat a stroke patient more than 200 kilometers away.

Radiomedix bets on alpha emitters for radiopharma’s future

In a small Houston clinic, more than a decade ago, patients with advanced cancer were receiving treatments that used radioactive molecules to seek out tumors and destroy them from within using an approach that would eventually help reshape oncology. Much of that early work was led by Radiomedix founder and CEO Ebrahim Delpassand, a nuclear medicine physician who later founded Radiomedix to translate those clinical insights into new drugs. Today, as radiopharmaceutical therapies surge into the spotlight with multibillion-dollar acquisitions and a growing pipeline of targeted radiation treatments, Radiomedix is marking its 20th anniversary while positioning itself to help define the field’s next chapter.

INHBE target scrutinized after Wave fallout

Wave Life Sciences Inc. wasn’t the only company to feel the stock market reaction to data from the phase I portion of its first-in-human Inlight trial testing 250 mg of WVE-007, an INHBE GalNAc-siRNA prospect, in otherwise healthy overweight or obese adults. Leerink analyst Mani Foroohar said in a report on Arrowhead Pharmaceuticals Inc. that the outcome of the trial by Wave “deflates [his] optimism” about with regard to INHBE. Another company in the space, Ibio Inc., saw its shares (NASDAQ:IBIO) drop about 28% on the WVE-007 news, though shares seem to have started a recovery.

FDA should do more to protect adcoms from conflicts, GAO says

With all the focus of late on the U.S. CDC’s Advisory Committee on Immunization Practices (ACIP), the FDA’s 30-plus advisory committees have been flying under the radar, especially since many of them haven’t met for a few years now. But that didn’t stop the Government Accountability Office (GAO) from digging into how the FDA handles potential conflicts of interest on its adcoms. In a congressionally mandated report released this week, the GAO looked beyond industry ties to point out the potential for both financial and “appearance” conflicts. It’s a lesson that could benefit the ACIP, as well.

Small-molecule TREM2 agonist advances to treat Alzheimer’s

Microglia play a central role in the neuroinflammation associated with Alzheimer’s disease (AD). These cells act as the brain’s immune system and respond to damage signals such as amyloid accumulation. When the process starts, the initial microglial response can be protective. However, in later stages, this response becomes dysfunctional and contributes to disease progression. At the 20th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD), held from March 17 to 21, 2026, in Copenhagen, scientists focused on TREM2, a microglial receptor that regulates immune responses, exploring new ways to address neuroinflammation. A small-molecule TREM2 agonist is emerging as a promising new approach to treat AD.

Novo’s weekly insulin Awiqli makes FDA grade

Novo Nordisk A/S scored U.S. FDA approval of Awiqli (insulin icodec) injection 700 units/mL, the first and only once-weekly, long-acting basal insulin. The drug is to be used as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Previously cleared in Europe and 13 other countries, the firm’s product has met difficulties in the U.S. that included an unfavorable advisory committee meeting in May 2024 and a complete response letter that summer.

Phase III wins lift shares of Compass, Palvella and Bridgebio in February

BioWorld tracked 152 clinical trial readouts across phases I through III in February 2026. Positive results from the second of two phase III trials have put Compass Pathways plc’s synthetic psilocybin therapy, COMP-360, on track for potential U.S. FDA approval within a year as the first classic psychedelic for treatment-resistant depression. Shares of the London-based company (NASDAQ:CMPS) rose 31% to close at $7.63 on Feb. 17.

Also in the news

3D Systems, Achieve Life Sciences, Agilent, Alzamend Neuro, Averna, Brainsway, Braveheart, Clemann Group, Concertai, Corcept, Crossject, Deepecho, Denali, Eos Senolytix, Faron, Forx, GC, Geron, GH Research, Hookipa, Humacyte, Icecure, Immunitybio, Invivoscribe, Io, Kodiak Sciences, Ligand, Lifeward, Lunai Bioworks, Microbot Medical, Neotrail, Neurolief, Norgine, Ono, Oramed, Philips, Plus, Prokidney, Poxel, Pulmatrix, Rani, Rhythm, Rubedo Life Sciences, Sironax, Soligenix, Spinogenix, Wave Life Sciences