Fast Track to SGX for CTCL
The Food and Drug Administration has granted Fast Track designation to SGX301 for the first-line treatment of patients with cutaneous T-cell lymphoma (CTCL).
SGX301, made by Soligenix Inc., is a novel first-in-class photodynamic therapy with the active ingredient of synthetic hypercin, a potent photosensitizer that has shown anti-proliferative effects on activated normal human lymphoid cells and has inhibited the growth of malignant T-cells isolated from CTCL patients. The drug is topically applied to skin lesions and then activated by fluorescent light 16 to 24 hours later.
The Fast Track designation, established under the FDA Modernization Act of 1997, is designed to facilitate frequent interactions with the FDA review team to expedite clinical development and submission of a New Drug Application for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. The designation permits the drug developer the opportunity to submit sections of an NDA on a rolling basis as data become available, allowing the FDA to review those materials on a rolling basis as well.
SGX301 had previously received Orphan Drug designation, which-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. This designation does not, though, shorten the duration of the regulatory review and approval process.
A Phase III clinical trial for the drug is expected to begin this year, according to a Soligenix news release.
Ibrutinib Approved for Waldenstrom's Macroglobulinemia
The Food and Drug Administration has expanded the approved use of Imbruvica (ibrutinib) for the treatment of patients with Waldenstrom's macroglobulinemia. The drug, co-marketed by Pharmacyclics and Janssen Biotech, works by blocking the enzyme that allows the abnormal B-cells in Waldenstrom's macroglobulinemia to grow and divide.
The approval-which was announced more than two months ahead of its prescription drug user fee goal date of April 17, the date the FDA was scheduled to complete review of the drug application-highlights the importance of development of drugs for supplemental indications, Richard Pazdur, MD, Director of the FDA's Office of Hematology and Oncology Products, said in a news release. "Continued research has discovered new uses of Imbruvica."
The drug had previously received a breakthrough therapy designation for the treatment of Waldenstrom's macroglobulinemia (OT 3/25/13 issue), and it is also currently approved for the treatment of patients with mantle cell lymphoma who have received one prior therapy (OT 12/10/13 issue) and patients with chronic lymphocytic leukemia who carry a deletion in chromosome 17 (OT 3/10/14 issue and 8/25/14 issue).
Imbruvica's most recent approval is based on a clinical study of 63 previously treated patients with Waldenstrom's macroglobulinemia, all of whom received a daily 420 mg orally administered dose until disease progression or side effects became intolerable. A total of 62 percent of the patients had their cancer shrink after treatment, and at the time of the study, the duration of response ranged from 2.8 months to approximately 18.8 months.
The most common side effects associated with the drug are thrombocytopenia, neutropenia, diarrhea, anemia, fatigue, musculoskeletal pain, bruising, nausea, upper respiratory tract infection, and rash.
Orphan Drug Status to Tarextumab from Pancreatic Cancer and SCLC
In addition, Orphan Drug designation was given to tarextumab (anti-Notch2/3, OMP-59R5) for the treatment of both pancreatic and small cell lung cancers. Tarextumab, made by OncMed, is a fully human monoclonal antibody that targets the Notch2 and Notch3 receptors. Preclinical studies have suggested that the drug works by downregulating Notch pathway signaling, causing anti-cancer stem cell activity and by affecting pericytes, impacting the stromal and tumor microenvironments.
The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
OncMed is currently enrolling patients in two trials for the drug: (1) a randomized Phase II clinical trial of tarextumab with gemcitabine plus Abraxane (paclitaxel-bound particles for injectable suspension, albumin bound) in patients with first-line advanced pancreatic cancer; and (2) a randomized Phase II clinical trial of tarextumab in combination with etoposide and platinum-based chemotherapy for patients with first-line extensive stage small cell lung cancer.
Phase Ib clinical and biomarker data from a study of tarextumab in combination with standard of care for patients with pancreatic cancer showed that the drug was well tolerated, side effects were manageable, and that 21 of 29 patients had a partial response or whose disease remained stable. A separate Phase Ib study of tarextumab in combination with etoposide and platinum chemotherapy in patients with small cell lung cancer showed that the combination was well tolerated and that 13 patients (out of the 16 who were evaluable for efficacy) had a partial response and for the three other patients disease was stable.
QIDP Designation to Catheter Lock Solution Neutrolin
The FDA designated Neutrolin Catheter Lock Solution as a Qualified Infectious Disease Product (QIDP) for oncology, hemodialysis, and intensive care unit patients, for whom catheter-related blood stream infections and clotting can be life-threatening.
The product, made by CorMedix, is a novel formulation of taurolidine, citrate, and heparin 1,000 u/ml that provides a combination preventative solution to decrease the triple threat of infection, thrombosis, and biofilm to keep catheters operating safely and efficiently by optimizing catheter blood flow while minimizing infections and biofilm formation.
The QIDP designation makes a drug eligible to benefit from certain incentives as provided under the Generating Antibiotic Incentives Now (GAIN) program, including FDA priority review, eligibility for Fast-Track status, and, if approved by the FDA, for an additional five-year extension of Hatch-Waxman patent exclusivity.
In order to achieve QIDP designation, a drug product must be intended to treat serious or life-threatening infections, particularly those infections caused by "qualified pathogens," as determined by the FDA. These pathogens include Staphylococcus aureus, Streptococcus species and Pseudomonas species, among others.
Neutrolin has shown antimicrobial activity against many of these qualified pathogens, several of which are known to pose a serious threat to the public health by causing blood-stream infections in hemodialysis, oncology, and intensive care patients, according to a news release from CorMedix.
The drug had previously been granted Fast Track designation, and Phase III trials are being planned.