Roche Holding AG received CE Mark approval for its Elecsys Apolipoprotein E4 (ApoE4) biomarker test, an in vitro diagnostic immunoassay to identify the ApoE4 gene variant from a blood sample. With APOE4 considered as the strongest genetic risk factor for developing Alzheimer’s disease, the test provides a fast, reliable way to determine whether an individual carries the genetic variant without the need for DNA‑based genotyping.
Rare disease drug development companies battle with economic challenges and small patient populations, but new technology alongside a human connection are helping researchers and marketers identify patients, educate physicians and build networks. At Pharma USA, a Reuters Events meeting in Philadelphia March 16-17, executives from Astrazeneca plc and UCB SA discussed their successes with rare disease launches, presenting their playbooks for building trust and driving adoption among patients.
Low expectations for Rhythm Pharmaceuticals Inc.’s phase III study dubbed Emanate with Imcivree (setmelanotide) in a handful of rare genetic obesities meant hardly any share price consequence, and the stock (NASDAQ:RYTM) closed March 17 at $87.68, down $2.83. “We’re not going to file [for approval] on any of these,” CEO David Meeker said, but the latest findings together represent “a solid building block going forward,” he added.
In one fell swoop March 16, a U.S. federal judge stayed the CDC’s January memo revising the childhood vaccine schedule and the Advisory Committee on Immunization Practices (ACIP) as reconstituted by Health and Human Services Secretary Robert Kennedy, along with everything that committee has done since early June.
Mestag Therapeutics Ltd. has raised a $40 million round from existing investors as it completes final preparations for its lead program MST-0312 to enter clinical development in mid-2026. MST-0312 is a bispecific antibody aimed at the lymphotoxin-β receptor (LTBR), which is essential for the development of lymphoid tissues.
Newco R1 Therapeutics Inc. has launched with an oversubscribed $77.5 million series A, providing the means to take AP-306, a potentially first-in-class hyperphosphatemia therapy through phase IIb development in patients with chronic kidney disease.
G2Gbio Inc. secured a ₩20 billion (US$13.35 million) investment from Samsung Epis Holdings Co. Ltd. and a joint research and license agreement for two assets, including a long-acting obesity treatment, with subsidiary Samsung Bioepis Co. Ltd. March 16.
Regulatory snapshots for biopharma and med tech, including global submissions and approvals, and other regulatory decisions and designations: Aminex, GSK, Mineralys.