United Therapeutics heads back to the FDA -- Gazette.Net



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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 FDA’s rejection in October.

At the time, the FDA said it had concerns that the drug, called oral trepostinil, didn’t 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 agency’s concerns came as a surprise, Jeffs said, as they hadn’t 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.”



In other Maryland bioscience industry news:

Claiming patent infringement, Sucampo Pharmaceuticals and its affiliated companies and partners are suing a California company and its partners for seeking regulatory approval to produce a generic version of their constipation drug, Amitiza.

In the suit, filed last week in U.S. District Court in Delaware, the Bethesda drug-maker, along with R-Tech Ueno and Takeda Pharmaceutical, said the application filed by Anchen Pharmaceuticals of Irvine and Par Pharmaceuticals infringes on six patents involving Amitiza; those patents are due to expire from 2020 to 2027.

Sucampo won marketing approval from the Food and Drug Administration for Amitiza in 2006 for 24 mcg capsules and in 2008 for 8 mcg capsules. In 2011, U.S. sales of Amitiza reached $226.4 million, according to the company’s latest annual filing with the Securities and Exchange Commission.

By suing, any FDA approval of Anchen’s application is automatically stayed for up to 30 months from Jan. 2, the date when Sucampo received Anchen’s notice that it was filing its application.

The plaintiffs want the court to block Anchen from receiving FDA marketing approval before their patents expire.

“Sucampo’s patent estate for Amitiz is strong, and the company intends to vigorously defend our intellectual property,” Thomas J. Knapp, Sucampo’s executive vice president and chief legal officer, said in a statement. “We believe that the Amitiza patents, granted by the U.S. Patent and Trademark Office, are valid, enforceable and infringed by Anchen’s proposed lubiprostone products.”

The defendants did not respond to a request for comment.



A Gaithersburg biotech has won orphan drug status from the FDA for its candidate to help treat brain cancer.

Lentigen’s product, called LG631-CD34, is designed for bone marrow protection in treating glioblastoma multiforme.

The new FDA designation qualifies Lentigen for seven years of market exclusivity following marketing approval, plus development-related incentives.

“The market exclusivity which this designation provides, together with our intellectual property rights, gives us increased impetus to advance the development of this product, enabling us to play our part in improving the lives of patients with glioblastoma,” CEO Tim Ravenscroft said in a statement.

The compound is now in a phase 1 clinical trial at University Hospitals Case Medical Center of Cleveland, supported by the National Institutes of Health.



RegeneRx Biopharmaceuticals was granted a patent allowance from Chinese regulators for using thymosin beta 4 to treat, prevent, inhibit or reduce heart tissue damage from heart failure disease.

The patent is to expire July 26, 2026, according to a statement from the Rockville biotech.



Supernus Pharmaceuticals, which last week launched sales of Oxtellar XR, reported this week that the epilepsy drug is being manufactured by Patheon of Mississauga, Ontario.

The Rockville drug-maker entered into the contract with Patheon in August, in anticipation of receiving FDA marketing approval, which it won in October, according to a new Supernus filing with the SEC.



The Maryland Stem Cell Research Commission has received 171 applications for funding, including five from Maryland biotechs that are ready to conduct preclinical trials.

Those trials would involve using stem cell products to treat HIV/AIDS, spinal cord injuries and demyelinating diseases such as transverse myelitis, according to information from the commission. Grants for these projects would be for up to $500,000 each.

The commission does not disclose the names of applicants, according to a spokeswoman.

Also, four applicants want funding to help support collaborations with researchers at the California Institute of Regenerative Medicine. In its two previous years, the states’ collaboration yielded three grants to Maryland scientists that leveraged $9.1 million in funding from California.

“We are pleased with the continued high level of interest in our groundbreaking research program, particularly by the number of applications from biotech companies ready to launch in-state preclinical trials,” Margaret Conn Himelfarb, chairwoman of the commission, said in a statement. “We are also delighted that several of our applicants are interested in collaborating with California researchers conducting clinical as well as basic research. Clinical trials are the gold standard, and this more advanced phase of investigation brings us a critical step further down the pipeline toward the patient.”



Cytomedix of Gaithersburg reported its Angel technology, used for preparing platelet-rich plasma, has been approved by Australian regulators and will be marketed and distributed in that nation by Medtel, a supplier of medical equipment and devices there.

The system is already marketed in Europe and the Middle East.

“Sales in international markets are a meaningful contributor to overall Angel revenues and are growing at an encouraging rate,” CEO Martin Rosendale said in a statement.

The plasma gel produced by the technology is used to treat wounds.



A Missouri company has acquired Cylex, a Columbia company that develops in vitro diagnostics used to illuminate immunity in transplant medicine.

Viracor-IBT Laboratories of Lee’s Summit did not disclose the price.

Cylex’s main product, ImmuKnow, helps physicians improve immunosuppression in recipients of kidney, liver, lung and heart transplants, according to a Viracor statement.

Viracor speicalizes in infectious disease, immunology and allergy testing. It is majority owned by a private equity firm, Ampersand Capital Partners.

rrand@gazette.net