FDA News Roundup: AstraZeneca, Pfizer, AbbVie, Actavis, And More
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
What FDA decisions did you miss this week?
Orphan Drug Designations
AstraZeneca’s MEK inhibitor selumetinib was named an orphan drug for uveal melanoma. The drug, which inhibits the MEK pathway and halts tumor growth, is currently being investigated in combination with chemotherapy in a Phase 3 trial enrolling first-line uveal melanoma patients. There are also trials underway investigating the drug’s effect on patients with KRAS mutation-positive lung cancer, thyroid cancer, and pediatric neurofibromatosis type 1.
RXi’s Samcyprone earned orphan drug designation for patients who have developed cutaneous metastases caused by stage 2b to stage 4 malignant melanoma. The treatment, a topical formulation of diphenylcyclopropenone, stimulates the immune system to tackle warts, alopecia areata, and cutaneous metastases. The drug is being developed as a safer, more consistent formulation of diphenylcyclopropenone , which has been used to treat skin conditions for several decades.
Oncolytics was primarily seeking orphan drug designation for its reolysin for pediatric patients with malignant glioma, however, following review, the FDA granted reolysin orphan designation for patients of all ages. In three brain cancer trials, the intravenous drug demonstrated its ability to infect brain tumors.
Ultragenyx’s triheptanoin (UX007) was awarded an orphan drug designation for the treatment of fatty acid oxidation disorders (FAOD). In the U.S., the drug is already considered an orphan drug for glucose transporter type-1 deficiency syndrome (Glut1 DS). Triheptanoin is a synthetic triglyceride compound being investigated in two Phase 2 trials, one for Glut1 DA and one for FAOD. The company has further goals of examining the drug’s potential at treating Glut1 DS-associated movement disorders.
Novogen and CanTx have received orphan designation for their ovarian cancer candidate, Cantrixil. A cyclodextrin envelop, Cantrixil is injected into the peritoneal and pleural cavities to halt the spread of tumors. The drug is intended to be a first-line therapy for early-stage ovarian, uterine, colo-rectal, and gastric carcinomas, with clinical trials launching in late 2015 to early 2016 for patients suffering from ovarian-cancer-associated malignant ascites.
FDA Approves Minocin sNDA
The supplementa New Drug Application (sNDA) for the injectable formulation of tetracycline derivative Minocin received FDA approval for the treatment of hospital-acquired infections, such as bacterial pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP). The company also received a Qualified Infectious Disease Product designation for the drug for HABP and VABP, infections in cystic fibrosis patients caused by Burkholderia cepacia complex or Stenotrophomonas maltophilia, and Burkholderia cepacia-caused pneumonia/lung infections in chronic granulomatous disease patients.
Seattle Genetics Hodgkin Lymphoma Drug Granted Priority Review
The FDA has accepted Seattle Genetics’ supplemental Biologics License Application (sBLA) for Adcetris for priority review. The treatment is indicated for the post-transplant consolidation treatment of Hodgkin lymphoma patients that are high risk for relapse or disease progression. The drug is currently being investigated in 30 clinical trials for Hodgkin lymphoma and systemic anaplastic large cell lymphoma (sALCL), as well as other CD30-positive malignancies.
FDA Expands Botox Label
Actavis’ Botox is now indicated for the treatment of upper limb spasticity in adults. In two trials, the drug was injected into the flexor pollicis longus and adductor pollicis thumb muscles, and was found to be effective in reducing thumb flexor muscle tone.
Pfizer Xalkori Wins Breakthrough Designation
Pfizer’z Xalkori earned FDA breakthrough therapy designation for those with ROS1-positive non-small cell lung cancer (NSCLC). The drug is currently approved for those with anaplastic lymphoma kinase (ALK)-positive metastatic lung cancer. The drug underwent investigation in an expansion cohort of a global Phase 1 study enrolling 50 NSCLC patients.
FDA Gives Priority Review To AbbVie HCV Regimen
AbbVie’s NDA for its genotype 4 (GT4) hepatitis C drug has been granted priority review. The drug is a combination of ombitasvir, paritaprevir, ritonavir (OBV/PTV/r), with ribavirin (RBV). The combination was previously awarded breakthrough therapy designation last summer. Enanta and AbbVie teamed up in 2006 to collaborate on HCV NS3 and NS3/4A protease inhibitors and drug combinations containing HCV protease inhibitors. Enanta’s contribution to this breakthrough therapy is Paritaprevir, which AbbVie is currently developing and commercializing. Paritaprevir has also been included in AbbVie’s treatment regimens for GT1 HCV.