FDA News

FDA grants orphan drug designation to cancer vaccine for rare bone tumor

The FDA granted orphan drug designation to the novel cancer vaccine BN-Brachyury for the treatment of chordoma, according to the agent’s manufacturer.

Chordoma is a rare type of cancer — affecting one in one million people per year — that develops along the spine and may present at the sacrum, mobile spine or the clivus.

BN-Brachyury (Bavarian Nordic) — a novel cancer immunotherapy vaccine — consists of a prime (MVA-BN) and a booster (fowlpox or FPV) dose, which are modified to express brachyury, a tumor-associated antigen that is overexpressed in some solid tumors, including chordoma.

Bavarian Nordic initiated a phase 1 trial earlier this year to assess the safety and tolerability of BN-Brachyury in up to 10 patients with metastatic or unresectable, locally advanced malignant solid tumors. Secondary endpoints include immunologic responses, measured by an increase in brachyury-specific T cells and other tumor-associated antigens.

Later this year, the company plans to initiate a phase 2 trial of up to 25 patients to assess the efficacy of BN-Brachyury in combination with radiation therapy. Overall response rate will serve as an endpoint.

“We are very happy to obtain orphan status for our chordoma program, given the severe lack of effective therapies for this population of patients,” Paul Chaplin, president and CEO of Bavarian Nordic, said in a company-issued press release. “Chordoma is a unique tumor [in which] the brachyury protein is universally overexpressed; which presents us with an exceptional opportunity to use our vaccine in a optimally targeted manner. We plan to initiate our phase 2 study in the fall of this year and to rapidly progress this program through proof of concept.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

The FDA granted orphan drug designation to the novel cancer vaccine BN-Brachyury for the treatment of chordoma, according to the agent’s manufacturer.

Chordoma is a rare type of cancer — affecting one in one million people per year — that develops along the spine and may present at the sacrum, mobile spine or the clivus.

BN-Brachyury (Bavarian Nordic) — a novel cancer immunotherapy vaccine — consists of a prime (MVA-BN) and a booster (fowlpox or FPV) dose, which are modified to express brachyury, a tumor-associated antigen that is overexpressed in some solid tumors, including chordoma.

Bavarian Nordic initiated a phase 1 trial earlier this year to assess the safety and tolerability of BN-Brachyury in up to 10 patients with metastatic or unresectable, locally advanced malignant solid tumors. Secondary endpoints include immunologic responses, measured by an increase in brachyury-specific T cells and other tumor-associated antigens.

Later this year, the company plans to initiate a phase 2 trial of up to 25 patients to assess the efficacy of BN-Brachyury in combination with radiation therapy. Overall response rate will serve as an endpoint.

“We are very happy to obtain orphan status for our chordoma program, given the severe lack of effective therapies for this population of patients,” Paul Chaplin, president and CEO of Bavarian Nordic, said in a company-issued press release. “Chordoma is a unique tumor [in which] the brachyury protein is universally overexpressed; which presents us with an exceptional opportunity to use our vaccine in a optimally targeted manner. We plan to initiate our phase 2 study in the fall of this year and to rapidly progress this program through proof of concept.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.