October 25th 2024
On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.
October 23rd 2024
In the complete response letter, the FDA classified deficiencies in the Abbreviated New Drug Application for ketamine as MINOR.
October 17th 2024
Following the approval of sodium oxybate for cataplexy in adults with cataplexy, the FDA approves this indication for the pediatric population.
The FDA has extended its review of the sNDA, delaying the previous October 15, 2024, PDUFA date, without providing a new anticipated action date.
October 15th 2024
The Accure Laser System is indicated for long term treatment of patients known to have mild to severe inflammatory acne vulgaris.
The Potential Role of Dupilumab in COPD Management, with Antonio Anzueto, MD
Antonio Anzueto, MD, discusses how dupilumab fits into treatment algorithms for patients with uncontrolled COPD.
Stephanie Christenson, MD: An "Exciting" New Era in COPD Management
Stephanie Christenson, MD, discusses the impact of recent regulatory approvals and pipeline movement for chronic obstructive pulmonary disease.
FDA Approves Ustekinumab (STELARA) Biosimilar for Chronic Inflammatory Diseases
The ustekinumab biosimilar will be available in early 2025, with indications to treat psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis.
Alnylam Submits sNDA for Vutrisiran for Treatment of ATTR-CM
The sNDA submission to the FDA included a Priority Review Voucher, which commits the regulatory agency to an accelerated review timeline.
FDA Grants Breakthrough Therapy Designation to Volixibat for Cholestatic Pruritus in PBC
The designation was granted to Mirum Pharmaceuticals based on a positive interim analysis of the phase 2b VANTAGE study of volixibat.
FDA Issues Second CRL for Dasiglucagon (Zegalogue) in Congenital Hyperinsulinism
The FDA's decision to issue a CRL was announced on October 08, 2024 and cites issues with the timing of the facility inspection.
FDA Grants Breakthrough Therapy Designation to Survodutide for Noncirrhotic MASH
In addition to the Breakthrough Therapy Designation, Boehringer Ingelheim announced the initiation of 2 phase 3 trials in adults with MASH and fibrosis and compensated MASH cirrhosis.
FDA Approves 3 Tubes of Aminolevulinic Acid Hydrochloride for Actinic Keratosis
The decision by the FDA follows an announcement by Biofrontera that there were no safety issues found following the submission of the medication’s supplemental New Drug Application.
5 FDA Decisions to Watch in Q4 2024
A review of 5 highly anticipated FDA decisions expected by year-end, including acoramidis for ATTR-CM, olezarsen for FCS, sotagliflozin for T1D and CKD, and more.
FDA Approves Exact Sciences’ Cologuard Plus Test for Colorectal Cancer Screening
The approval is based on findings from BLUE-C, where Cologuard Plus demonstrated sensitivities of 95% for colorectal cancer and 43% for advanced precancerous lesions at 94% specificity.
Aldeyra Resubmits New Drug Application for Reproxalap for Dry Eye Disease
The resubmission includes positive data from a recently completed dry eye disease symptom trial requested by the FDA after a review of the previous NDA.
Denifanstat Receives Breakthrough Therapy Designation for Noncirrhotic MASH
The designation was based on positive data from the phase 2b FASCINATE trial of denifanstat in noncirrhotic MASH with moderate to advanced fibrosis.
TransCon hGH Aims for FDA Label Expansion for Adult Growth Hormone Deficiency
Ascendis Pharma has submitted a sBLA to the FDA for TransCon hGH for the treatment of adults with growth hormone deficiency.
FDA Approves Dupilumab (Dupixent) for Treatment of COPD
This approval by the FDA follows positive findings from the pivotal BOREAS and NOTUS trials on adults with uncontrolled COPD.
FDA Approves Xanomeline and Trospium Chloride (Cobenfy) for Schizophrenia
The FDA approves Cobenfy, the first pharmacological approach to treating schizophrenia since the 1970s.
FDA Approves Levacetylleucine for Niemann-Pick Disease Type C
Announced on September 24, 2024, the approval marks the second treatment approved by the FDA for Niemann-Pick disease type C within one week.
FDA Accepts sNDA for Roflumilast Foam as Scalp, Body Psoriasis Treatment
The FDA has set a PDUFA target action date of May 22, 2025, for roflumilast foam 0.3% for individuals aged ≥12 with scalp and body psoriasis.
FDA Accepts NDA for Delgocitinib Cream Treatment of Chronic Hand Eczema
The FDA decision to accept the new drug application for delgocitinib cream is also occurring alongside the drug’s acceptance by the European Commission.
FDA Approves Arimoclomol, First Drug for Niemann-Pick Disease, Type C
Arimoclomol, in combination with miglustat, is approved to treat neurological symptoms associated with NPC in adults and children aged ≥2 years.
FDA Issues CRL to Vanda Pharmaceuticals’ Tradipitant for Gastroparesis Symptoms
The FDA declined to approve the NDA for tradipitant for the treatment of symptoms of gastroparesis, suggesting that Vanda conduct additional studies.
FDA Approves Benralizumab for Eosinophilic Granulomatosis with Polyangiitis
Announced on September 18, 2024, the approval of benralizumab for adults with EGPA, which is based on the MANDARA trial, marks the second FDA approval for EGPA.
FDA Clears Eversense 365, Marking First 1-Year CGM Option in Diabetes
The FDA has approved the Eversense 365, the world’s first 1-year continuous glucose monitor, with US availability expected in late 2024.
FDA Warns of Liver Injury Risk from Fezolinetant for Hot Flashes
The FDA advises stopping the fezolinetant treatment at the first signs of liver injury to prevent further damage and help restore normal liver function.
Nanoscope Reports Positive End-of-Phase 2 Meeting for MCO-010 Gene Therapy
Nanoscope Therapeutics announced the meeting with the FDA supported the advancement of MCO-010 into Phase 3 for Stargardt disease.
FDA Approves Lebrikizumab Treatment of Eczema Among Patients Aged 12 and Older
Eli Lilly’s announcement of the lebrikizumab-lbkz approval for patients 12 years of age and older follows positive findings from the ADvocate 1, ADvocate 2, and ADhere studies.
FDA Approves Dupilumab for Adolescents with Chronic Rhinosinusitis with Nasal Polyps
This approval expands the drug’s age range and follows recent positive phase 3 findings on dupilumab treatment for patients aged 12 to 17 with CRSwNP.
FDA Gives Authorization for First Over-the-Counter Hearing Aid Software Device
This marketing authorization by the FDA allows the Hearing Aid Feature to be implemented alongside compatible Apple AirPod Pro headphones.
FDA Grants Breakthrough Therapy Designation to Plozasiran for FCS
The agency's designation was granted to plozasiran as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.
FDA Approves Guselkumab (Tremfya) for Ulcerative Colitis
The decision is based on rates of clinical and endoscopic remission with guselkumab in the ongoing phase 2b/3 QUASAR study.
FDA Approves Travere Therapeutics’ Sparsentan to Slow Kidney Function Decline in Adult Primary IgAN
The FDA has granted full approval to sparsentan (Filspari) for slowing kidney function decline in adults with IgAN at risk of disease progression based on PROTECT trial data.