FDA News Roundup: Baxter, Celgene, Agios, And More
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
What FDA decisions have you missed recently?
Orphan Drug Designations
GlycoMimetics’ e-selectin antagonist AbbVie’s Humira has earned an orphan drug designation for moderate-to-severe hidradenitis suppurativa, known as Hurley Stage 2 and Stage 3 disease. The FDA is currently reviewing a supplemental Biologics License Application (sBLA) to add the HS indication to its already lengthy list of indications.
Diurnal’s Infacort, an oral formulation of hydrocortisone, was named an orphan drug for pediatric Adrenal Insufficiency (AI). To date, children with the condition have had to take treatments compounded from adult tablets, which can lead to issues including early puberty, virilisation, and infertility. The pediatric formulation is currently in Phase 3 trials in Europe, with results expected in 2016.
Alnylam Pharma’s candidate revusiran, an RNAi therapy for transthyretin (TTR)-mediated amyloidosis, earned orphan drug designation. The drug is also being investigated in clinical trials for patients with Familial Amyloidotic Cardiomyopathy (FAC) and TTR cardiac amyloidosis.
Two candidates from PlasmaTech Biopharma earned orphan drug designation for Sanfilippo Syndromes A and B. The company also earned Rare Pediatric Disease designation for both candidates, which grants the candidates priority review vouchers.
Aura Biosciences won orphan designation for AU-011 for uveal melanoma — a condition which currently has no approved therapies on the market. The treatment contains tumor-targeting nanoparticles that are joined to a laser-activated molecule. In animal testing, three administrations of AU-011 eliminated tumors completely and spared the retina.
Fast-Track Designations
Luspatercept has been fast tracked for both transfusion-dependent and non-transfusion dependent beta thalassemia. Celgene and Acceleron are both working to launch a Phase 3 clinical program for the treatment this year. This treatment is a modified activin receptor type IIB fusion protein responsible for red blood cell precursor cell differentiation and maturation. Apart from beta-thalassemia, the treatment is currently undergoing investigation in a Phase 2 trial for patients with myelodysplastic syndromes.
Agios’ AG-120 for acute myelogenous leukemia (AML) was awarded Fast Track designation. The drug is indicated specifically for those with an isocitrate dehydrogenase-1 mutation and is currently being investigated in two Phase 1 studies—one for solid tumors and the other for blood diseases. In the future, the company plans to partner with Celgene on a Phase 3 study investigating the IDH1-inhibitor in AML patients.
Immunomedic’s Sacituzumab govitecan was given Fast Track designation for patients with non-small-cell lung cancer (NSCLC). The antibody-drug conjugate is indicated as a third-line treatment for patients who have not seen success with targeted therapies including ALK inhibitors, EGFR inhibitors, and PD-1 inhibitors. Results from a mid-stage trial show that the treatment demonstrated promise in treating breast, lung, esophageal, colorectal, and bladder cancers.
Baxter Saline Manufacturing Facility Given Thumbs Up
The FDA has granted the company approval to manufacture 0.9 percent Sodium Chloride Injection at the Sabinanigo, Spain facility. This approval for production of 250 mL, 500 mL, and 1000 mL of saline will help the company address shortages in the U.S.
Johnson & Johnson Wins FDA Approval
Schizophrenia drug, Invega Trinza, is indicated to be used four times a year. The injection can only be taken following four months of treatment with the once-monthly treatment Invega Sustenna. In a long-term maintenance trial, the newly approved treatment demonstrated the ability to eliminate any significant schizophrenia symptoms in 93 percent of patients.