New FDA Orphan Drugs: Gestiva, Onconase, Aerosolized Ciprofloxacin

Yael Waknine

February 26, 2007

February 26, 2007 — The US Food and Drug Administration (FDA) has granted orphan drug status for a long-acting injectable form of a naturally occurring progesterone in the prevention of recurrent preterm birth; ranpirnase for the treatment of malignant mesothelioma; and an inhaled liposomal formulation of ciprofloxacin for the management of bronchiestasis.


Orphan Drug Progesterone Injection (Gestiva) in the Prevention of Recurrent Preterm Birth

On January 31, the FDA granted orphan drug status for a long-acting injectable form of a naturally occurring progesterone known as 17 alpha-hydroxyprogesterone caproate (Gestiva, made by Adeza) for the prevention of preterm birth in women with a history of preterm delivery.

Preterm birth is defined as delivery before 37 weeks of gestation. Citing the New England Journal of Medicine (NEJM) in a news release, the company notes that preterm births have historically accounted for up to 85% of all pregnancy-related complications and deaths in the United States. Women with a history of preterm birth represent one of the highest risk groups for a future preterm delivery.

The approval was based in part on data from a multicenter, double-blind, placebo-controlled trial (n = 463) showing that weekly injections of the long-acting progesterone formulation significantly reduced the preterm birth rate by 34%. Furthermore, infants born to women in the active treatment group had significantly decreased rates of necrotizing enterocolitis, intraventricular hemorrhage, use of supplemental oxygen, and mean duration of respiratory therapy.

The recommended dose of the long-acting progesterone formulation is weekly intramuscular 250 mg injection between 16 weeks 0 days (160 weeks) and 20 weeks 6 days (206 weeks) of gestation until 37 weeks of gestation or birth.

According to a company news release, obstetrician/gynecologists began using the product in women with a history of preterm birth based on a 2003 American College of Obstetricians and Gynecologists (ACOG) recommendation that followed a National Institutes of Health study published in June 2003 in the NEJM. However, the formulation is prepared solely by compounding pharmacies at this time.

A request for the drug's priority review for approval is currently under consideration by the FDA; if approved, it will be the only such FDA-approved product studied by the NIH and recommended by ACOG for the prevention of recurrent preterm birth.


Orphan Drug Ranpirnase (Onconase) for Malignant Mesothelioma

On January 30, the FDA approved orphan drug status for ranpirnase (Onconase, made by Alfacell Corp) in the treatment of malignant mesothelioma.

According to a company news release, ranpirnase has demonstrated the ability to target tumor cells while sparing normal cells in both in vivo and in vitro studies. Incorporated into the cytosol of malignant cells by endocytosis, the agent selectively degrades cytosol transfer RNA (tRNA), thereby inhibiting protein synthesis, stopping cell proliferation, and inducing apoptosis.

Ranpirnase therapy for malignant mesothelioma is currently being evaluated in a confirmatory phase 3b study involving more than 50 sites in the United States, Canada, Europe, New Zealand, and Australia. Other potential indications under investigation include non–small cell lung cancer and other solid tumors.

Ranpirnase was previously granted orphan drug status for this indication in the European Union and Australia.


Aerosolized Ciprofloxacin Product Granted Orphan Drug Status for Bronchiestasis

On January 16, the FDA approved orphan drug designation for an inhaled liposomal formulation of ciprofloxacin (made by Aradigm Corp) in the management of bronchiestasis.

According toa company news release, bronchiestasis is a relatively rare condition that affects about 110,000 people in the United States and results from a cycle of inflammation, recurrent infection, and bronchial-wall damage. Most patients require recurrent or chronic antibiotic treatment to suppress the lung infection, which is often due to Pseudomonas or other bacteria.

Designed to prolong the short-acting nature of ciprofloxacin and localize its activity to the lungs, the aerosolized formulation is intended to allow sufficient pulmonary exposure to treat these infections.

The formulation was previously granted orphan drug status by the FDA in April 2006 for the management of cystic fibrosis.

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