Gazette.Net: United Therapeutics heads back to the FDA

Four months after the Food and Drug Administration shot down United Therapeutics application to market a tablet version of its drug for pulmonary arterial hypertension, the Silver Spring company has reapplied to the regulatory agency.

United Therapeutics, which already sells intravenous, subcutaneous and inhaled versions of its major money-maker, has been working for years to gain regulatory approval for an oral version.

United Therapeutics stock rose 5.5 percent Thursday morning on the news; it had tumbled 15.3 percent following the FDAs rejection in October.

At the time, the FDA said it had concerns that the drug, called oral trepostinil, didnt show enough improvement in patients who took it in clinical trials, as measured by how far they could walk in six minutes. The agency suggested that another phase 3 trial should possibly involve fixed and more frequent dosing, such as three times a day versus twice daily, COO Roger Jeffs said in a conference call then.

Some of the agencys concerns came as a surprise, Jeffs said, as they hadnt been raised in regulators discussions with the company.

Since then, company officials have met with regulators to gain a better understanding of their concerns, and the resubmitted application addresses those concerns, United Therapeutics spokesman Andrew Fisher said Thursday. Obviously, no new trials have been conducted since then.

The FDA has categorized United Therapeutics resubmission as a class 1 review, Fisher said. Such reviews tend to entail minor new analyses of existing data, whereas class 2 reviews involve new data and as such have longer review periods.

The company expects to hear back from the FDA by the end of March; class 2 reviews typically run six months, he said.

The company has projected sales of about $1 billion for the other forms of the drug this year, and with 30,000 Americans being treated for pulmonary arterial hypertension, the oral version shows much sales promise, CEO Martine Rothblatt said during the October call.

The potential market of about 20,000 patients for oral trepostinil represents luscious and delectable low-hanging fruit, Rothblatt said. Most companies would salivate to have (the oral version) in their pipeline.

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Gazette.Net: United Therapeutics heads back to the FDA