FDA News Roundup: Amgen, Janssen, Actavis, And More

Amgen BLA Up For FDA Review

Amgen’s BLA for its cholesterol drug evolocumab was accepted by the FDA for review. Evolocumab is responsible for stopping a key protein, proprotein convertase subtilisin/kexin type 9 (PCSK9), which often halts the removal of “bad cholesterol” from the blood. The drug’s safety and efficacy was investigated in 10 Phase 3 studies enrolling 6,800 patients who continued to have high cholesterol regardless of statin/other prior treatment, patients who couldn’t tolerate statins, and those with homozygous familial hypercholesterolemia (HoFH). A decision from the FDA on the application is expected by August 27, 2015.

Threshold Pharma TH-302 Snags Fast Track Designation

An investigational anticancer drug, TH-302, received Fast Track designation for patients with metastatic or locally advanced unresectable soft tissue sarcoma (STS). The drug, a hypoxia-activated prodrug, is currently undergoing a Phase 3 trial under a special protocol assessment with the FDA. TH-302 targets tumor hypoxia regions in tumor cells — regions believed to play a key role in the formation of cancer drug resistance. The company expects to have a better picture of the drug’s effect on overall survival in early 2016. Of the 640 patients enrolled in the study, one group is receiving TH-302 in combination with doxorubicin, while the other group is receiving only doxorubicin. Overall survival is the primary endpoint, with secondary endpoints being progression-free survival, overall response rate, pharmacokinetics, and safety.

RedHill Bio Drug Granted QIDP Status

RHB-105, a Phase 3 Helicobacter pylori (H. pylori) eradication therapy, received Qualified Infectious Disease Product (QIDP) designation last week for those suffering from H. Pylori-linked chronic gastritis, peptic ulcer disease, and gastric cancer. The drug has already demonstrated positive results in a Phase 2 trial in Australia, with results from an ongoing Phase 3 study expected mid-2015. RHB-105 is in capsule form and contains two antibiotics and a proton pump inhibitor (PPI). The company hopes that the drug will eventually be approved to treat a larger group of patients, included those with an active or recent history of ulcers, as well as those who do not or have not had any ulcers. Should the FDA approve RHB-105, it would be the first drug indicated to treat this larger patient population with H. Pylori infection.

Polyphor Antibiotic Awarded QIDP Designation

POL7080 was granted QIDP designation for those with Pseudomonas aeruginosa-linked Ventilator-Associated Bacterial Pneumonia (VABP). The drug, which belongs to a new class of antibiotics targeting Gram-negative bacteria, was examined in a Phase 1 clinical trial and is currently in Phase 2 trials enrolling VABP or Bronchiectasis patients. Polyphor has been working with Roche since 2013 on the development and commercialization of POL7080.

Xeris Pharma’s HI Drug Wins Orphan Drug Designation

The FDA granted Orphan Drug Designation to Xeris Pharmaceuticals’ soluble glucagon, indicated for the prevention of hypoglycemia in those with congenital hyperinsulinism (HI). The drug also received Orphan Designation from the European Commission for the treatment of HI. The drug could offer patients a new treatment option for a disease that has often been treated by removing the pancreas — a move that makes many patients permanently reliant on insulin for the rest of their lives. The company’s glucagon formulation was designed to be stable at room temperature and to be delivered via pump so that administration is continuous and can prevent severe hyperglycemia.

Janssen sNDA For Schitzoaffective Disorder Gets FDA Nod

Janssen’s sNDA for Invega Sustenna received a nod of approval from the FDA late last week, enabling the drug to be used as a monotherapy or adjunctive therapy to treat schizoaffective disorder. The drug is taken once a month and is a long-acting antipsychotic that was investigated in a 15-month long-term maintenance trial to determine its ability to delay schizoaffective disorder relapse. This is the first drug approved by the FDA to be a monotherapy for the treatment of schizoaffective disorder.

Actavis’ Saphris Faces FDA Review

Actavis’ sNDA for bipolar medication Saphris (asenapine) has been accepted for priority review by the FDA, with a decision expected in the beginning of 2015. The drug is indicated to treat pediatric patients 10 to 17 years of age suffering from manic or mixed episodes associated with bipolar disorder. The drug was studied in a 3-week monotherapy trial in which 302 patients out of a total 403 received asenapine twice daily in fixed doses of 2.5mg, 5 mg, and 10 mg. The drug was successful in reducing both the Young Mania Rating Scale (YMRS) total score and Clinical Global Impression-Bipolar (CGI-BP) score when compared to placebo.

Sanofi’s Lemtrada Wins FDA Approval For MS

Sanofi’s Genzyme announced the FDA’s approval of Lemtrada as a treatment for multiple sclerosis nearly a year after the agency rejected it for security concerns last December. While it has been approved, the company acknowledges that, because of its safety profile, the drug might best serve those who have had inadequate responses to other current standards of care for MS. Administered intravenously, the drug is taken in two treatment rounds: the first lasts 5 days and then, after a year has passed, another round is administered over the course of 3 days. While there are several other non-injectable treatment options on the market from Novartis and Biogen Idec, Sanofi’s Genzyme hopes that the fact the drug can reduce the risk of relapse will encourage patients to choose Lemtrada. According to Reuters, analysts expect the drug to bring in roughly $400 million by 2018.

Genentech Avastin Approved For Additional Indication

Genentech’s Avastin has garnered yet another approval from the FDA, this time for the treatment of ovarian cancer. The drug is to be taken in combination with chemotherapy for women with platinum-resistant, recurrent ovarian cancer. In the Phase 3 AURELIA study, the drug reduced the risk of disease progression and death in patients also receiving chemotherapy involving paclitaxel, pegylated liposomal doxorubicin, or topotecan. The drug is currently indicated for the treatment of brain, colon, lung, cervical, and kidney cancer, and in 2013, brought Roche roughly $6 billion in revenue.