Drug Pipeline News

The indication is for patients 12 years and older hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

The FDA has approved the first generic once-daily GLP-1 injection for the improvement of glycemic control for type 2 diabetes in patients 10 years and up.

The expanded indication is approved to reduce excess body weight and maintain reduction long-term in children 2 years and up with obesity due to BBS, POMC, or LEPR deficiency.

The primary endpoint was non-inferiority in HbA1c levels after 26 weeks.

With the federal agency's decision, remestemcel-L-rknd has become the first FDA-approved mesenchymal stromal cell therapy.

From new topical dermatology treatments for atopic dermatitis to the first nasal spray to treat type 1 allergic reactions, these are our top FDA approvals of 2024.

Currently, golimumab is approved in adults with moderately to severely active ulcerative colitis.

The federal agency has set a target action date of June 10, 2025 for potential approval.

Arcutis seeks FDA approval for roflumilast cream 0.05% (ZORVYE) to treat mild to moderate atopic dermatitis in children 2 to 5 years.

The FDA has approved Crenessity for managing classic CAH in patients 4 years and up, reducing steroid doses while controlling hormones, offering a safer treatment option.

The halt follows a severe respiratory disease safety signal observed in a July 2024 phase 1 trial of Moderna's mRNA-1345 and mRNA-1365 vaccine candidates.

Pharming Group N.V. plans to include these findings in worldwide regulatory filings in 2025.

The submission to treat PsO is for children aged 6 years and up while the jPsA submission is to treat children aged 5 years and older.

Both monthly migraine days and headache days were reduced vs placebo in patients aged 6 to 17 years.

101-PGC-005 is Type IA prodrug of dexamethasone that targets CD206+ macrophages.

If approved, SL1009 has the potential to be the first approved medication for PDCD, and would be available as an oral solution.

Currently, there are no treatments for the serious, rare pediatric disease.

Once-daily icotrokinra versus placebo demonstrated clinically significant skin clearance in those with moderate-to-severe plaque psoriasis.

Results demonstrated that daily oral infigratinib treatment led to increases in annualized height velocity and improvement in body proportionality.

With this expanded FDA clearance, the wearable becomes the first FDA-cleared, non-drug therapy to treat acute migraines in children.

The application indication is for adults and pediatric patients aged 12 years and older with CSU whose disease is not adequately controlled with H1 antihistamines.

With the decision, Kebilidi becomes the first FDA-approved gene therapy for AADC deficiency, and is indicated for adult and pediatric patients.

A target action date for potential approval of prademagene zamikeracel (pz-cel) has been set for April 29, 2025.

Data stems from the first 4 patients dosed in a low-dose cohort of an ongoing phase 1/2 open-label trial.

The initiation of a phase 2 study of the investigational 31-valent pneumococcal conjugate vaccine is expected by the end of January 2025.

The federal agency has not raised any concerns regarding safety and efficacy of tapinarof cream, 1%.

Application acceptance was based on positive results with monthly and bi-monthly dosing data from a phase 3 trial.

The approval makes methotrexate, currently, the only only oral liquid methotrexate on the market approved for both adult and pediatric indications.

The fibrin sealant solution is now available in 18 countries.

Look back at several quick video interviews discussing the expanding role of artificial intelligence, how RSV treatment has evolved, and much more.