Intellia Therapeutics’ lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to the swelling attacks from the rare disease hereditary angioedema. Intellia has filed a regulatory submission for this genetic medicine, which could become the first in vivo gene-editing therapy to land FDA approval.

The post Intellia’s Data Reveal Tees Up FDA Filing for CRISPR-Based In Vivo Gene-Editing Med appeared first on MedCity News.

Merck’s HIV drug Idvynso could become a drug choice for patients who need to switch from Gilead Sciences’ Biktarvy for safety or tolerability reasons. The Merck combination drug pairs two molecules that bring different mechanisms of action to suppressing HIV.

The post FDA Nod for Merck HIV Drug Brings Competition to a Gilead Sciences Antiretroviral appeared first on MedCity News.

Travere Therapeutics’ Filspari is now the first FDA-approved drug for focal segmental glomerulosclerosis (FSGS), a rare kidney disease. Analysts project the pill will become a blockbuster seller.

The post Bouncing Back From Trial Failure, Travere Wins First FDA Approval in Rare Kidney Disease appeared first on MedCity News.

FDA approval for Eli Lilly’s oral GLP-1 drug, Foundayo, comes about three months after Novo Nordisk launched its Wegovy pill. While clinical trial results for both daily pills showed comparable weight reduction, Lilly’s pill offers patients a slight dosing advantage.

The post Eli Lilly Gets Speedy FDA Nod for Oral GLP-1 Drug, a Competitor to New Novo Nordisk Pill appeared first on MedCity News.

March has seen a slew of executive hires, exits and layoffs across the healthcare industry. For instance, Flare Capital Partners, SCAN Group and Advocate Health named new executives. There were also layoffs at organizations including Oracle, Takeda and Blue Shield of California.

The post Healthcare Moves: A Monthly Summary of Hires, Exits and Layoffs appeared first on MedCity News.

For consumers and clinicians the difference between “FDA-cleared,” “FDA-registered,” and general wellness products is not always obvious. Clear communication about intended use and supporting evidence is essential for maintaining trust.

The post Red Light Therapy’s Regulatory Implications appeared first on MedCity News.

Denali Therapeutics’ Avlayah received FDA approval for treating Hunter syndrome, a rare neurological disorder. The biologic medicine uses Denali’s proprietary drug delivery technology to cross the protective blood-brain barrier.

The post FDA Approval for Denali Therapeutics Blazes a New Trail for Brain-Penetrating Drugs appeared first on MedCity News.

Aldeyra said the FDA did not ask for another clinical trial for reproxalap in dry eye disease, but there is a “lack of substantial evidence” that warrants exploration about the reasons for trial failures to identify the appropriate patients for the eye drop. AbbVie holds an option to collaborate on reproxalab’s commercialization.

The post Lacking Evidence, FDA Rejects Aldeyra Dry Eye Disease Drug appeared first on MedCity News.

Leucovorin is now approved for cerebral folate deficiency months after FDA Commissioner Marty Makary claimed the decades-old generic drug had promise for treating autism. The FDA’s review was based on published literature and real-world evidence.

The post FDA Drug Approval Marks a First for a Disease — But It’s Not Autism appeared first on MedCity News.

The FDA said consolidating safety reporting into a single platform, the Adverse Event Monitoring System (AEMS), will increase transparency and reduce costs. But like the legacy systems it replaces, AEMS reports are unverified so causation and frequency of events cannot be determined.

The post FDA Transparency Push Expands to Monitoring Safety of Vaccines and Other Regulated Products appeared first on MedCity News.

Povetacicept, a Vertex Pharmaceuticals immunology drug designed to block two targets, met the main goal of a Phase 3 clinical trial in the rare kidney disease immunoglobulin A nephropathy. The fusion protein came from Vertex’s acquisition of Alpine Immune Sciences, and analysts say the drug has best-in-class potential.

The post Vertex Pharma to Seek Speedy FDA Approval for Kidney Disease Drug From $4.9B Acquisition appeared first on MedCity News.

Ampreloxetine’s Phase 3 failure in multiple system atrophy is the third disappointing clinical trial outcome for the Theravance Biopharma drug. The biotech will now wind down all R&D and explore options that could include the sale of the company.

The post Theravance Drug Fails Another Pivotal Test, This Time Sparking Restructuring That Ends R&D appeared first on MedCity News.

The label expansion for Boehringer Ingelheim’s Hernexeos was made under an FDA pilot program that accelerates regulatory review of products with national interest. The daily pill is now approved as a first-line treatment for HER2-positive non-small lung cancer, an indication with few treatment options.

The post FDA Voucher Leads to Speedy Approval of Boehringer Drug for First-Line Use in Lung Cancer appeared first on MedCity News.

Despite missing statistical significance, Grail said the observation of trending improvement suggests potential for better results with more time, so the company is extending follow up by up to a year. More detailed trial results will be submitted for presentation at the annual meeting of the American Society of Clinical Oncology.

The post Grail’s Galleri Blood Test Falls Short in Large Cancer-Screening Study, Shares Sink 50% appeared first on MedCity News.

The FDA asked for more data to determine whether Disc Medicine’s bitopertin is benefiting patients with erythropoietic protoporphyria, a rare blood disorder. Analysts say it’s a surprising delay considering bitopertin is one of the first drugs in a new FDA pilot program that speeds up regulatory review of medicines deemed critical for national security or public health.

The post Disc Medicine Rare Disease Drug Picked for Faster Regulatory Review Is Rejected by the FDA appeared first on MedCity News.

PTC Therapeutics said it cannot resolve differences it has with the FDA in interpreting clinical trial data for Translarna, a drug it developed for Duchenne muscular dystrophy. The rare disease drug developer’s decision to withdraw Translarna’s FDA submission comes nearly a year after a negative European regulatory decision for the drug.

The post PTC Therapeutics Ends Bid to Seek FDA Approval for Rare Muscle Disease Drug appeared first on MedCity News.

The FDA’s refuse-to-file letter cited no safety or efficacy concerns for Moderna’s influenza vaccine, mRNA-1010. The company said the agency’s justification for refusing a review is inconsistent with guidance given prior to the start of Phase 3 testing.

The post Moderna Says FDA Refusal to Review mRNA Flu Vaccine Contradicts Federal Rules, Prior Guidance appeared first on MedCity News.

Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe.

The post Clinical Trial Holds Cast Doubt on Approval Chances of Regenxbio Gene Therapy for Rare Disease appeared first on MedCity News.

Tenpoint Therapeutics’ Yuvezzi brings a combination approach to presbyopia compared to single mechanisms of action offered by once-daily eye drops from Orasis Pharmaceuticals, Lenz Therapeutics, and AbbVie. Yuvezzi’s commercialization will be supported by up to $235 million in financing.

The post Presbyopia Eye Drop From Startup Tenpoint Gets FDA Approval, Paving Way for $235M Financing appeared first on MedCity News.

A report supporting the CDC’s revisions says the U.S. is an outlier in the number of diseases and the total number of vaccine doses recommended in its pediatric vaccine schedule. But some physician groups say comparisons to other countries can be misguided and removing vaccines from the schedule puts children’s health at risk.

The post CDC Removal of Six Diseases From Child Vaccination Schedule Sparks Concern From Clinicians appeared first on MedCity News.

December has seen a slew of executive hires, promotions and layoffs across the healthcare industry. For instance, Breyer Capital, the FDA and Strive Health named new executives. There were also layoffs at organizations including Alameda Health System, Pfizer and Voyager Therapeutics.

The post Healthcare Moves: A Monthly Summary of Hires, Exits and Layoffs appeared first on MedCity News.

Omeros’s complement system drug Yartemlea is now FDA approved for treating a severe and potentially fatal complication of hematopoietic stem cell transplants. Our recap of other regulatory actions includes drug approvals in cancer, rare disease, and metabolic disorders.

The post Fatal Complication of Stem Cell Transplants Gets Its First FDA-Approved Therapy appeared first on MedCity News.

Beyond weight reduction and long-term maintenance of weight loss, the FDA decision for oral Wegovy covers use of the once-daily pill for reducing the risk of major cardiovascular events. Novo Nordisk expects the Wegovy tablet will launch in early January.

The post Novo Nordisk’s Wegovy Pill Becomes First FDA-Approved Oral GLP-1 Drug for Weight Loss appeared first on MedCity News.

Cytokinetics drug Myqorzo won FDA approval for treating obstructive hypertrophic cardiomyopathy, or oHCM. Compared to Bristol Myers Squibb’s projected blockbuster drug Camzyos, the Cytokinetics daily pill has less restrictive safety and monitoring requirements, giving it a competitive advantage, according to analysts.

The post FDA Approval Puts New Cytokinetics Heart Drug in Competition With Fast-Growing BMS Med appeared first on MedCity News.

GSK’s Exdensur requires just two injections a year to treat severe asthma. Less frequent injections give this new biologic medicine a dosing edge over one of GSK’s own blockbuster respiratory products as well as severe asthma drugs from companies such as AstraZeneca, Sanofi, and Amgen.

The post GSK Gets FDA Approval for New Severe Asthma Drug With a Dosing Advantage appeared first on MedCity News.

Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology.

The post FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder appeared first on MedCity News.

GSK submitted the same Blenrep clinical trial data to global regulators and the FDA, but received a slightly more limited regulatory approval in the U.S. In an interview during the annual meeting of the American Society of Hematology, GSK oncology executive Hesham Abdullah explained what else the FDA wants from Blenrep clinical trials.

The post At ASH, GSK’s Top Cancer Exec Discusses the FDA’s Different Take on Multiple Myeloma Drug Blenrep appeared first on MedCity News.

Pharvaris’s deucrictibant met the main and secondary goals of its Phase 3 test as an on-demand treatment for swelling attacks caused by the rare disease hereditary angioedema. Pharvaris’s capsule could compete against Kalvista Pharmaceuticals’ Ekterly, an HAE pill approved by the FDA over the summer.

The post Pharvaris Drug for Rare Swelling Disease Meets Phase 3 Goals; FDA Filing Planned for 2026 appeared first on MedCity News.

The accelerated FDA approval of Bayer’s Hyrnuo covers treatment of advanced cases of non-small cell lung cancer with HER2 mutations. It will compete against an HER2-targeting Boehringer Ingelheim pill approved in August.

The post Bayer Lung Cancer Drug Lands Speedy FDA Nod to Stay Competitive With a Boehringer Ingelheim Med appeared first on MedCity News.

Arrowhead Pharmaceuticals’ RNAi drug plozasiran, brand name Redemplo, is now the second FDA-approved therapy for familial chylomicronemia syndrome, an inherited lipid disorder. Beyond its claims of safety and dosing advantages, Arrowhead set a dramatically lower price for its first commercial product, which will compete against an Ionis Pharmaceuticals drug.

The post With FDA Approval for RNAi Drug, Arrowhead Pharma Starts Price War With Rival Ionis appeared first on MedCity News.