FDA Roundup: Sanofi, Genentech, Apitope, Retrophin, And More
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
What regulatory decisions have you missed recently?
Antibiotic Approved For New Pediatric Age Group
The antibiotic meropenem was approved for complicated intra-abdominal infections in infants under the age of 3 months. An NIH-run trial provided proof that the antibiotic — currently approved for abdominal and skin infections in children 3 months+ and adults — was effective in the younger age group.
Reformulated Antiretroviral Approved For Children’s Use
The FDA recently approved a new formulation of HIV antiretroviral lopinavir and ritonavir, manufactured by Cipla. The antiretroviral is now available in heat stable oral pellets that can be easily mixed into food, making it easier for children to take the medication.
Orphan Drug Designations
Aptose Biosciences’ APTO-253 won orphan designation for acute myeloid leukemia (AML). Currently in a Phase 1b clinical trial for AML, the treatment previously demonstrated its abilities to destroy AML cells as a monotherapy or in combination with other candidates in preclinical trials. AML is often the result of the suppression of the Kruppel-like factor 4 (KLF4), and as such, APTO-253 is designed to induce the KLF4 tumor suppression gene.
An oral product from Lipocine, LPCN 1107, was named an orphan drug to prevent preterm birth. The oral hydroxyprogesterone caproate (HPC) candidate performed well in a Phase 1b clinical trial. However, as there is another competitor’s candidate named an orphan drug and already approved for this indication, Lipocine needs to prove that the drug is clinically superior to earn exclusivity for this condition.
Isarna Therapeutics received orphan designation for its locked nucleic acid-modified antisense oligonucleotide ISTH0036 for those experiencing scarring after glaucoma filtration surgery. The treatment is currently being investigated in a Phase 1 clinical trial.
Lion Biotechnologies’ LN-144, an immunotherapy based on tumor-infiltrating lymphocytes, was named an orphan drug for malignant melanoma, stage 2b to stage 4. The company anticipates launching a Phase 2 clinical trial later this year.
Arrowhead Research’s Alpha-1 Antitrypsin deficiency-associated liver disease treatment, ARC-AAT, is currently being studied in patients with PiZZ genotype AATD enrolled in a Phase 1b clinical trial. A Phase 1a trial enrolling healthy volunteers has already been completed.
Apitope’s preclinical ATX-F8-117 for hemophilia A has earned orphan drug designation. The treatment is currently indicated for patients with hemophilia A that possess or are at risk of developing inhibitors.
MediciNova’s MN-166 was awarded orphan drug designation for Krabbe disease. The company is currently putting together an IND application for an FDA review for the candidate. MN-166, or ibudilast, is also indicated for the treatment of post-stroke complications and bronchial asthma in Japan and Korea. It is also undergoing investigation for MS and other neurological conditions.
Edge Therapeutics’ EG-1962 won orphan designation for aneurysmal subarachnoid hemorrhage (aSAH). The candidate is a polymeric nimodipine microparticle that can be applied to the injury site.
AM Pharma’s recombinant human Alkaline Phosphatase, or recAP, garnered orphan designation for hypophosphatasia. The treatment is currently being investigated in a Phase 2 trial for acute kidney injury. The company also has found that, in mouse models, recAP improved bone and teeth mineralization, prevented seizures and ectopic calcification, and kept craniosyntosis symptoms at bay.
Sanofi Wins Breakthrough Therapy Designation
Genzyme’s treatment for non-neurological effects of Niemann-Pick disease type B, olipudase alfa, was awarded breakthrough therapy designation. The disease, marked by a buildup of sphingomyelin, does not have any approved treatments. This treatment replaces the enzyme acid sphingomyelinase, which is not sufficiently produced in those with the disease, and in turn, enables the breakdown of excessive amounts of sphingomyelin. So far, the treatment has passed through a Phase 1 clinical trial enrolling five patients, and the company plans to launch a Phase 2/3 trial later this year.
Actemra Wins Breakthrough Designation
Genentech’s Actemra for systemic sclerosis, or scleroderma, is quick on the path to becoming the first approved therapy for the rare disease. The treatment is currently under investigation in a Phase 3 trial for systemic sclerosis. The designation was supported by results from a Phase 2 faSScinate study showing the drug cut down on skin thickening.
PKAN Treatment Put On Fast Track
Retrophin’s RE-024, a phosphopantothenate replacement therapy candidate, was fast tracked for patients with pantothenate kinase-associated neurodegeneration (PKAN). The company is currently selecting patients for a Phase 1 trial to determine the drug’s safety and tolerability. There are currently no approved treatments on the market for PKAN.
FDA Committee Recommends Praluent And Repatha
The first PCSK9-targeting injection for cholesterol is on the brink of earning approval. The FDA committee has voted favorably on Praluent’s benefits, observed in the ODYSSEY Phase 3 program enrolling 5,000 patients. The drug when taken with a statin is capable of lowering cholesterol in patients who have not had success with statins and other cholesterol-lowering treatments alone. However, some panelists have come out in support of approving it solely for those with familial hypercholesterolemia. Similarly, others are concerned about the drug’s effect on cardiovascular health, which will not be known until large-scale studies are completed in 2017.
The committee also came out in support of Amgen’s competitor in the cholesterol space, Repatha. By a unanimous vote, the committee supported the approval of the drug for those with familial hypercholesterolemia, and an 11 to 4 vote believes the drug should be made available to patients who have not been sufficiently treated with statins.