Azitra Inc ($AZTR) announced an update on their ongoing clinical study. Azitra Inc. is running a first-in-human study called “A Randomized, Double-Blind, Vehicle-Co... In a crowded dermatology field with players in atopic and autoimmune skin care, a focused rare-disease asset may help Azitra stand out, though commercial scale will depend on follow-on trials and partner interest. This clinical study has been completed and recently updated, with fuller details and status available on the ClinicalTrials portal. The model is randomized and blinded to body side, aiming mainly to assess treatment effects rather than prevention or diagnosis. The trial was first submitted in November 2023, marking the formal launch of the program for this asset. The latest update was filed on July 27, 2026, signaling that data and documentation have been refreshed as the study reached completion and moved toward analysis. For investors, a completed early-stage study in a rare disease can support a higher risk appetite toward AZTR if safety looks clean and benefit signals appear strong. Azitra Inc. is running a first-in-human study called “A Randomized, Double-Blind, Vehicle-Controlled, First-in-Human Safety, Tolerability and Proof-of-Concept Study of Topical ATR12-351 in Adults With Netherton Syndrome.” The trial tests if ATR12-351 is safe and shows early benefit in adults with this rare skin disorder, which could open a new niche in dermatology. The treatment is a topical drug called ATR12-351, an ointment designed to release a protective skin protein using a modified skin bacterium. Azitra Inc (AZTR) announced an update on their ongoing clinical study.

Original title: “Azitra’s ATR12-351 Trial Completion Puts Rare-Disease Skin Drug on Investors’ Radar - TipRanks.com”

FDA today licensed Ezplaz Freeze Dried Plasma (FDP), making it the first freeze-dried plasma product licensed for use in the United States. Ezplaz is intended for transfusion in adult patients who need plasma and for whom other plasma products are not available.

Original title: “ FDA Licenses First-Ever Freeze-Dried Plasma Product in the U.S. ”

Designation supports pivotal trials for emerging candidates

Original title: “Atsena gains EMA orphan status for two gene therapies”

'We now have the clinical data package to support global submissions for retatrutide as a potential treatment for obesity, knee osteoarthritis pain, and obstructive sleep apnea.' The results from the TRIUMPH-2 and TRIUMPH-3 studies provide a clinical data package to support global regulatory submissions for retatrutide as a potential treatment for obesity, knee osteoarthritis pain, and obstructive sleep apnea. The clinical data package will support global submissions for the drug as a potential treatment for obesity, knee osteoarthritis pain, and obstructive sleep apnea. “Across five positive phase 3 studies, retatrutide has shown powerful efficacy, and we believe it could be an important future tool in the management of cardiometabolic health,” says Kenneth Custer, PhD, executive vice president and president, Lilly Cardiometabolic Health, in a release. “With the positive results from TRIUMPH-2 and TRIUMPH-3, we now have the clinical data package to support global submissions for retatrutide as a potential treatment for obesity, knee osteoarthritis pain, and obstructive sleep apnea. In the TRIUMPH-3 trial, adults with severe obesity and established cardiovascular disease, with or without type 2 diabetes, lost up to an average of 55.8 lbs (22.6%) at 80 weeks on the highest dose. The study also found that major adverse cardiovascular events occurred less frequently in participants taking retatrutide compared to the placebo group.

Original title: “Phase 3 Trials Show Weight Loss for Triple Agonist with OSA Potential | Sleep Review”

The biologics faced headwinds last ... work on rare-disease drugs as necessary to maintain the agency’s high bar for approval. A tough FDA analysis: Briefing documents posted by the FDA ahead of Capricor’s advisory committee don’t appear kind to its cell therapy. But the debate will likely center on what statistical analysis should be used to evaluate the treatment and the timing of the unblinding of the underlying trial... The biologics faced headwinds last year under former FDA Commissioner Marty Makary and former Center for Biologics Evaluation and Research Director Vinay Prasad — both of whom publicly defended their work on rare-disease drugs as necessary to maintain the agency’s high bar for approval. A tough FDA analysis: Briefing documents posted by the FDA ahead of Capricor’s advisory committee don’t appear kind to its cell therapy. But the debate will likely center on what statistical analysis should be used to evaluate the treatment and the timing of the unblinding of the underlying trial. An FDA advisory committee will hold meetings this week to review applications for novel therapies disfavored by those who have since left the agency. The agency said the new recommendations stem from workshops with Friends of Cancer Research and the American Society of Clinical Oncology. Michael Severino is Sarepta Therapeutics’ new CEO. He previously led Tessera Therapeutics and worked at AbbVie. The FDA’s Pediatric Advisory Committee will meet on Sept. 16 to review post-marketing safety for more than two dozen drugs and devices. The FDA published four new case studies Monday demonstrating successful approaches by drugmakers that carried out rare disease drug development programs. CANCER CLINICAL TRIAL GUIDANCE — The FDA’s oncology center on Monday released three final guidance documents aimed at making it easier for more cancer patients to participate in clinical trials. “Fewer than 5% of cancer patients currently receiving treatment are enrolled in clinical trials even though more than 70% say they are willing to participate,” the FDA said in an email.

Original title: “FDA panel to consider previously spurned drugs - POLITICO”

The FDA's rebuff of Replimune comes a day after the agency similarly called into question the Phase 3 trial for Capricor’s Duchenne muscular dystrophy cell therapy.

Original title: “FDA says Replimune’s melanoma data package is ‘not interpretable,’ setting up tough adcomm”

First patient enrolled in pivotal coronary study

Original title: “OrbusNeich begins Japanese trial for drug‑coated balloon”

European regulators move closer to approving option for young male patients

Original title: “Nezglyal receives positive CHMP opinion supporting treatment access for boys with cALD”

In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor's assertion that a Phase 3 trial met its primary outcome measures.

Original title: “FDA disputes Capricor's claims of Phase 3 success for DMD cell therapy”

Otsuka Pharmaceutical plans to launch Simtriyo—the first norepinephrine, dopamine, serotonin reuptake inhibitor—later this year following scheduling by the U.S. Drug Enforcement Agency.

Original title: “Otsuka’s ADHD approval opens up new drug class with $615M opportunity”

The experimental anticancer therapy CR-001 was generally well tolerated in a first-in-human clinical trial that tested several doses in 11 people with acute myeloid leukemia (AML), a type of blood cancer. Following treatment, six of the 10 patients evaluable for effectiveness had stable disease, ... The experimental anticancer therapy CR-001 was generally well tolerated in a first-in-human clinical trial that tested several doses in 11 people with acute myeloid leukemia (AML), a type of blood cancer. Following treatment, six of the 10 patients evaluable for effectiveness had stable disease, meaning their cancer did not worsen. The modification is designed to make miR-15a more stable and help it enter cells, where it may reduce levels of proteins that support cancer growth and treatment resistance. Researchers conducted a first-in-human clinical trial to evaluate the therapy’s safety and tolerability in people with AML. The study enrolled 11 participants, most of whom had AML that had returned or failed to respond after multiple prior lines of therapy. Participants received CR-001 by infusion once weekly for four weeks at various doses. Those whose disease remained stable after the first four infusions could receive up to four additional weekly infusions. CR-001 was generally well tolerated in a first-in-human AML trial, with early disease control seen in six of 10 evaluable patients. Regulators in 2 countries OK clinical testing of new blood cancer treatment · Adding blood cancer treatment to AML care safe, effective in 2 early trials · Solu Therapeutics’ CMML treatment put on FDA fast track

Original title: “Experimental AML therapy shows early safety, disease control”

Twelve new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended twelve medicines for approval at its July 2026 meeting.The committee…, CHMP statistics Key figures from the July 2026 CHMP meeting are represented in the graphic below. © European Medicines Agency , CHMP statistics:…, Positive recommend

Original title: “Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 July 2026”