New FDA Orphan Drugs: MB07133, VitiGam, ALS-357

Jill Taylor

October 05, 2007

October 5, 2007 -- The US Food and Drug Administration (FDA) has granted orphan drug designation to MB07133 for the treatment of liver cancer, VitiGam for the treatment of stage IIB to stage IV metastatic melanoma, and ALS-357 for the topical treatment of metastatic melanoma.

Orphan Drug MB07133 for Liver Cancer

On September 10, Metabasis Therapeutics, Inc, announced that the FDA granted orphan drug designation to MB07133 for the treatment of hepatocellular carcinoma, the most common form of primary liver cancer.

Primary liver cancer originates in the liver itself. Although only approximately 18,000 new cases of primary liver cancer are diagnosed annually in the United States, hepatocellular carcinoma is worldwide the most common solid-organ tumor. At present, there is no FDA-approved therapy or standard of care for treatment.

MB07133, a novel prodrug of cytarabine monophosphate (araCMP), is designed to produce the oncolytically active form, cytarabine triphosphate (araCTP), in the liver tumor. Preliminary results from phase 1/2 clinical trials suggest that the drug is well tolerated and active against the disease, as evidenced by tumor shrinkage and disease stabilization.

Orphan Drug VitiGam for Stage IIB to Stage IV Metastatic Melanoma

On July 8, GammaCan International, Inc, announced that the FDA granted orphan drug designation to VitiGam for the treatment of stage IIB to stage IV metastatic melanoma, a form of skin cancer that is estimated to cause 75% of all skin cancer-related deaths.

Melanoma that is rapidly diagnosed can be effectively treated by surgical removal. However, the prognosis is poor for patients who are diagnosed with metastatic disease (stage III and stage IV), with a median survival time of 8.5 months. The most recent drug approval by the FDA for the treatment of metastatic melanoma took place over 30 years ago.

VitiGam is a first-in-class immunoglobulin G-based anticancer immunotherapy manufactured from the plasma of donors with vitiligo, a benign skin condition affecting pigmentation. Studies have shown that the agent contains potent antimelanoma activity.

Orphan Drug ALS-357 for Metastatic Melanoma

On August 28, the FDA granted orphan drug designation to ALS-357 (Advanced Life Sciences Holdings, Inc) for the topical treatment of metastatic melanoma.

Originating in melanocytes, a type of cell in the skin that produces the pigment that gives skin its natural color, melanoma can occur on any skin surface and become metastatic, spreading to other parts of the body. The disease is resistant to most of the currently available therapies and is commonly fatal.

ALS-357 is a novel drug that induces apoptosis, or programmed cell death, in the tumor cells. The agent has shown promise in both in vitro and in vivo preclinical studies and is currently entering phase 1/2 clinical development.

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