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FDA’s rare disease decisions are strongest when the patient community has a voice in advisory committee decisions.
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Psychedelics are gaining momentum in depression, with one treating physician predicting that the drug class could “wipe out the SSRIs” if safety and durability hold up.
Saol Therapeutics is the latest biotech to resubmit for approval of a drug rejected under former FDA Commissioner Marty Makary, following REGENXBIO and Replimune.
Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.
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Congressional letters sent to the CEOs of Eli Lilly, Pfizer, Merck, BMS and AbbVie this week voicing concerns about the pharmas’ clinical trials in China highlight an ongoing discrepancy in how government and industry think about the rise of the Asian country’s biotech industry.
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The acquisition of Aliada Therapeutics gives AbbVie access to a Phase I anti-amyloid antibody as well as the biotech’s novel platform engineered for efficient blood-brain barrier transport.
The Swiss pharma is paying $150 million upfront to gain rights to Monte Rosa’s VAV1-targeting molecular glue degraders, led by a Phase I candidate which holds therapeutic promise for immune-mediated diseases.
Jefferies analyst Kelly Shi in a Sunday note to investors said that both data drops for Revolution Medicines’ experimental RAS inhibitors are positive and could be “synergistic” in the first-line setting for pancreatic ductal adenocarcinoma.
Orlynvah is the first oral penem approved in the U.S. and Iterum Therapeutics’ first FDA-approved product. CEO Corey Fishman said the company will renew its efforts to look for a potential partner to maximize value for its stakeholders.
William Blair analyst Myles Minter in a Monday note to investors said that Vertex’s povetacicept “has maintained its potential to be a best-in-class asset” in the IgA nephropathy space and could become a “multibillion-dollar pipeline-in-a-drug product” for autoimmune disorders, while “outstanding questions” remain for Biogen’s felzartamab before moving into pivotal studies.
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.
Vertex may have pivoted away from the space, but candidates in development by Arrowhead/Takeda, Wave, Korro and others could address the damage underlying alpha-1 antitrypsin deficiency and make today’s treatments a thing of the past.
Pfizer CEO Albert Bourla is in a tough spot as activist investor Starboard Value continues to call for a change in the company’s leadership. However, analysts are supportive of the embattled executive.
The French drugmaker’s newly launched respiratory syncytial virus antibody Beyfortus far exceeded analyst expectations, bringing in almost $700 million in the third quarter. Leerink Partners analyst David Risinger in a Friday note to investors said Sanofi expects Q4 Beyfortus sales similar to Q3.