News Feature | August 11, 2014

What You Missed From The FDA Last Week—August 5-9, 2014

By Anna Rose Welch, Editor, Biosimilar Development
Follow Me On Twitter @AnnaRoseWelch

FDA Expands Lumizyme Approval For Younger Patients

The FDA expanded the approval of the drug Lumizyme for patients with infantile-onset Pompe disease to include patients under 8 years old. The rare genetic disorder causes heart and skeletal muscle weakness that leads to respiratory weakness and death for 1 in every 40-300,000 births. Lumizyme is a lysosomal glycogen-specific enzyme that the FDA approved back in 2010, however there were some restrictions to its use (Risk Evaluation and Mitigation Strategy (REMS)); only patients older than 8 were eligible for the treatment. Following this most recent approval, the REMS program will be eliminated.

FDA Designates AbbVie Compound An Orphan Drug

AbbVie’s investigational compound ABT-414 has received Orphan Drug Designation in the treatment of glioblastoma multiforme. The compound, an anti-epidermal growth factor receptor antibody drug conjugate is carried through the blood stream and releases its cytotoxic agent once it’s inside the targeted cancer cell. Apart from being in a phase 1 clinical program for patients with recurrent or unresectable glioblastoma multiforme, the drug is also being investigated as a treatment for squamous cell tumors.

FDA Accepts Jakafi NDA For Priority Review

The FDA has accepted Incyte’s Supplemental New Drug Application (sNDA) for Jakafi (ruxolitinib) for patients with rare blood cancer polycythemia vera (PV) who have not responded or responded insufficiently to hydroxyurea. The drug was investigated in the RESPONSE Phase 3 trial, which was conducted under a Special Protocol Assessment (SPA) from the FDA. The company has set an end goal Prescription Drug User Fee Act (PDUFA) date in early December. The drug is currently approved for the treatment of intermediate to high-risk myelofibrosis (MF).

Genoa Pharma Pirfenidone Treatment Granted Orphan Drug Designation

Genoa Pharmaceuticals’ inhaled GP-101 for the treatment of IPF received FDA orphan drug designation for the treatment of idiopathic pulmonary fibrosis (IPF). Oral pirfenidone (known as Esbriet) has shown potential in slowing the disease progression. However, the company emphasizes that treatment still requires a large oral dose to be effective, hence the new reformulation of the drug into this new aerosol form. 

The Medicine’s Company Orbactiv Approved By FDA

Orbactiv was approved as an injection for adults with acute bacterial skin and skin structure infections (ABSSSIs). This is the first antibiotic approved by the FDA for the treatment of ABSSSIs needing only a single, once-daily administration. This approval is based off of the SOLO I and SOLO II trials, which enrolled 1,987 patients, as well as a subset of 405 patients suffering from MRSA infection. The drug was found to be non-inferior compared to vancomycin. In 2012, the product garnered QIDP status under the GAIN act.

Ebola Virus Test Kit Given Green Light

The U.S. military received a thumbs up from the FDA to use its currently unapproved test-tube diagnostic test to identify the Zaire strain of Ebola, responsible for upwards of 2,500 infections and deaths in West Africa. The test is to be used for those who are demonstrating signs or symptoms of infection, or for those who have or may have been exposed. This approval was granted under a special emergency-use provision. Officials don’t expect an Ebola vaccine to be approved until 2015.

CTI Biopharma Granted Fast Track Designation For Pacritinib

Myelofibrosis treatment Pacritinib was granted fast-track designation. The drug, a novel JAK2 inhibitor, is intended for those with intermediate and high risk myelofibrosis, which can also include patients with disease-related or treatment-related thrombocytopenia. Pacritinib is currently being investigated in two phase 3 clinical trials in the PERSIST program for patients with myelofibrosis.

Lannet’s ANDA Approved By FDA

Lannet Company announced that the FDA granted its Abbreviated New Drug Application for Oxycodone Hydrochloride capsules (5mg) approval.

Pharma Talking Points

The FDA has released a list of updated user fee amounts for 2015 for the medical device, pharmaceutical, generic drugs, biosimilar drugs areas, as well as for drugs compounded at outsourcing facilities.

A new study published by Health Affairs suggests that the passage of the Prescription Drug User Fee Act (PDUFA) in 1992 has not only accelerated approvals of drugs, but may also be contributing to the increasing rate of Black Box warnings and product withdrawals.

According to the Wall Street Journal blog, the Department of Health and Human Services has received a request from several U.S. senators who have called upon the agency to announce when a formal policy for naming biosimilars will be launched. WHO recently released a proposal specifying a biosimilar naming policy, however, as it is optional, individual regulatory agencies from country to country are not required to comply with WHO’s proposal. The most recent letter from the U.S. senators did not argue in favor of any particular policy to follow for the naming of biosimilars.