Orphan drugs account 19 sales in 2020

Drug candidate shows clinical activity in phase II studies

The FDA’s Office of Orphan Products Development has granted orphan drug status to VAL-083 (DelMar Pharmaceuticals), a first-in-class small-molecule chemotherapeutic, for the treatment of women with ovarian cancer. VAL-083 previously received an orphan drug designation for glioma and medulloblastoma in the United States.

In more than 40 phase I and phase II clinical studies sponsored by the National Cancer Institute, VAL-083 demonstrated clinical activity against a range of malignancies, including lung, brain, cervical, and ovarian cancers and leukemia, both as a single agent and in combination with other treatments.

Ovarian cancers are commonly treated with platinum-based chemotherapy regimens. Initial tumor response rates are relatively high; however, up to 75% of ovarian cancer patients who respond to initial treatment will relapse within approximately 18 months after completing first-line therapy. Median survival in platinum-resistant recurrent ovarian cancer patients has ranged from six to nine months in published studies.

At the recent annual meeting of the American Association for Cancer Research, investigators at the University of Texas MD Anderson Cancer Center presented preclinical data demonstrating that the mechanism of action of VAL-083 appears to differ from that of the platinum-based chemotherapies currently used to treat ovarian cancer. In these studies, VAL-083 demonstrated an ability to circumvent cisplatin resistance in all ovarian cell lines tested.

The American Cancer Society has estimated that approximately 22,000 women receive a new diagnosis of ovarian cancer and approximately 14,000 die from the disease each year in the U.S. Ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. The market for ovarian cancer therapies is expected to be approximately $570 million in 2016 and is projected grow to more than $3.5 billion by 2022.

Source: DelMar Pharmaceuticals; April 21, 2016.

More Headlines

Use of Fluconazole During Pregnancy May Result in Miscarriage
FDA investigates study findings
Cabozantinib (Cabometyx) Wins FDA Nod for Patients With Advanced Renal Cell Carcinoma
Therapy improves overall survival, progression-free survival, and objective response rate in phase 3 trial
FDA Approves Bevespi Aerosphere for Patients With COPD
Treatment demonstrates superior improvement in lung function versus mono-components and placebo
FDA Panel Votes Against Muscular Dystrophy Drug Eteplirsen
Approval decision expected by May 26
Independent Analysis Shows Avoidable Hospital Deaths Remain High
Nonprofit group issues safety score update
FDA Accepts Resubmission of New Drug Application for IV Carbamazepine for Epilepsy
No IV formulations are available
New Infection Alert in Catheters Could Tackle Hospital “Superbugs”
System detects bacterial biofilms
Study shows 100% cure rate in GT1b patients without use of ribavirin
Heartburn Drugs Linked to Serious Kidney Disease
New data corroborate risk of PPIs

Our most popular topics on Managedcaremag.com