RDR Alert®

Crinecerfont significantly reduces glucocorticoid use in pediatric CAH while improving hormone levels, enhancing long-term patient outcomes.

FDA further postpones action date for elamipretide, a potential treatment for Barth syndrome, as discussions on labeling progress.

Our top 5 headlines spotlight FDA approvals for maralixibat and dupilumab, plus key trial results in diabetes, cardiovascular disease, and retinal vein occlusion.

The FDA cleared the expanded indication for the treatment of endogenous hypercortisolemia in adults with Cushing’s syndrome.

The FDA approval of diazoxide choline extended-release tablets (Vykat XR) was announced on March 26, 2025.

AstraZeneca announced eneboparatide normalized serum calcium with statistical significance at 24 weeks compared with placebo.

Our top 5 headlines featured an oral drug delivering 16% weight loss, notable blood pressure reductions from a hypertension treatment, and 3 key FDA decisions.

Chronic hypoparathyroidism requires holistic management addressing more than just calcium normalization to achieve optimal patient outcomes.

ATSN-201 gene therapy has previously received Rare Pediatric Disease and Orphan Drug designations from the regulatory agency.

With this historic approval, revakinagene taroretcel (ENCELTO) becomes the first and only FDA-approved treatment for MacTel.

The regulatory agency has assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2025.

Castells describes what’s missing from the current treatment landscape and the potential implications of elenestinib for indolent systemic mastocytosis.

Epioxa has been assigned a target action date of October 20, 2025, for the regulatory agency’s review of the NDA.

With this FDA approval, chenodiol tablets become the first approved drug to treat CTX, a rare lipid storage disease.

More than 90% of patients with hemophilia B eliminated factor IX prophylaxis 4 years after treatment with etranacogene dezaparvovec (HEMGENIX) gene therapy.

Once-weekly tirzepatide may be an alternative to daily hormone shots in patients with congenital generalized lipodystrophy.

Awarded to Beacon Therapeutics, RMAT designation allows enhanced communications with the FDA across the development of the investigational gene therapy.

Results from the Phase 3 LIBerate-HoFH trial found LDL-C responses generally similar between patients treated with lerodalcibep and evolocumab.

Axatilimab in 9 mg and 22 mg vial sizes are expected to launch in early February for the treatment of chronic graft-versus-host disease.

With a Phase 3 trial expected in 2025, linsitinib is the first oral small-molecule therapy to demonstrate statistical significance in thyroid eye disease.

All participants with RP treated with OCU400 gene therapy achieved vision improvement or preservation at 2 years of the Phase 1/2 trial.

Atumelnant rapidly lowered key biomarkers in congenital adrenal hyperplasia in positive topline results from the Phase 2 TouCAHn trial.

Additional TEASE-3 participants who completed 24 months of gildeuretinol therapy reported no progression in Stargardt disease, including stable visual acuity.

OCS-05 meets primary safety endpoints and demonstrates a neuroprotective structural benefit, as well as visual improvement, in topline results from ACUITY.

On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.