AZEDRA treats patients 12 and older with two different types of rare tumors — pheochromocytoma or pragaglioma — and is the first FDA-approved treatment of its kind.

Progenics Pharmaceuticals (NASDAQ:PGNX) announced on Monday (July 30) that the US Food and Drug Administration (FDA) approved the company’s rare tumor treatment.

The treatment, AZEDRA, was given the seal of approval from the FDA to treat patients aged 12 and older with rare tumors of the adrenal gland–either pheochromocytoma or paraganglioma–that requires anti-cancer therapy.

According to the FDA release, this is the first approved drug by the organization for this kind of therapy.

“As the first FDA approved therapy for unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy, AZEDRA provides a new treatment option for physicians and their patients,” Mark Baker, CEO of Progenics said in the release.

Baker continued, stating that AZEDRA had proven to decrease blood pressure medication requirements and even reduced the size of the tumor in some patients.

The FDA said the efficacy of the drug was demonstrated in a single-arm, open-label clinical trial with 68 patients. Roughly 17 patients experienced a reduction of 50 percent or more of all antihypertensive medications that lasted at least six months, while overall tumor response was found in 15 of those patients.

“Many patients with these ultra-rare cancers can be treated with surgery or local therapies, but there are no effective systemic treatments for patients who experience tumor-related symptoms such as high blood pressure,” Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said in the FDA release. “Patients will now have an approved therapy that has been shown to decrease the need for blood pressure medication and reduce tumor size in some patients.”

Pheochromcytoma and paraganglioma are tumors that arise in cells around the adrenal glans. These tumors commonly secrete high levels of hormones that often lead to these patients having life-threatening high blood pressure, heart failure and stroke.

That being said, there are of course serious side affects attached to AZEDRA because it is a radioactive therapeutic agent. Progenics says in the release effects can range from risk associated with radiation exposure, bone marrow-related issues, thyroid problems, elevations in blood pressure, kidney problems and respiratory problems.

The FDA says the drug can cause harm to a developing fetus and also that radiation exposure associated with AZEDRA can cause infertility in both men and women.

AZEDRA was granted by the FDA through fast track, breakthrough therapy and priority review designations. The FDA also granted Progenics orphan drug designation for AZEDRA, which aims to “assist and encourage the development” of drugs to treat rare diseases.

“The drug’s Fast-Track status and Breakthrough Therapy designation by the FDA underscores the dire need for the development and expeditious review of diagnostic and therapeutic agents for pheo/para that, generally, don’t get adequate prioritization despite the growing prevalence of these and other NET cancers globally,” Emily Collins, president of the Pheo Para Alliance said in Progenics’ press release.

Following the announcement on Monday, shares of Progenics spiked from its market open of $US7.63 to reach US$8.31 by 3:30 p.m. EST. By Tuesday (July 31), however, shares of the company dipped back down to US$8.06 as of 3:42 p.m. EST.

Progenics currently has a “Moderate Buy” ranking on TipRanks based off two analyst ratings with an average price target of US$15.50, a high of US$16 and low of US$15. Chad Messer, an analyst from the firm Needham, reiterated his “Buy” position on Progenics on Tuesday as a result of the company’s FDA approval.

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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.



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