Don't Forget To
Rate This Article

Get the Latest Investment Ideas Delivered Straight to Your Inbox. Subscribe


Pharma Co. Shares Get a Bang from FDA Orphan Drug Approval
Trending Company

Share on Stocktwits


Shares of C4 Therapeutics Inc. traded 16% higher after the biopharma company reported that the U.S. FDA granted Orphan Drug approval status for its CFT8634 for use in treating soft tissue sarcoma.

Clinical-stage biopharmaceutical company C4 Therapeutics Inc. (CCCC:NASDAQ) (C4T), which is focused on developing next generation small-molecule medicines that exhibit the potential to eliminate disease-causing proteins for use in treatments for cancer and other diseases, today announced that "the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CFT8634 for the treatment of soft tissue sarcoma."

The company explained that the FDA assigns ODD status to medicines and biologics used to address rare conditions or diseases that typically affect less than 200,000 people in the U.S. The firm noted that "ODD provides certain benefits, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval."

C4 Therapeutics' Chief Medical Officer Adam Crystal, M.D., Ph.D. remarked, "Patients living with synovial sarcoma currently have limited treatment options. After first-line treatment with chemotherapy, the benefit of which is typically of limited durability, patients with metastatic synovial sarcoma tend to do poorly."

"The FDA's decision to grant orphan drug designation to CFT8634 is an important recognition of the potential of our targeted protein degrader to address this dire unmet medical need faced by patients and their families," Dr. Crystal added.

The company described CFT8634 as "a BiDAC™ degrader targeting BRD9 for the treatment of cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1 deleted cancers."

The firm stated that until recently, BRD9 was believed to be an "undruggable" target as bromodomain inhibitors of BRD9 were not proven to be effective in treating these cancers. C4 Therapeutics advised that "its TORPEDO® platform was leveraged to discover CFT8634, an orally bioavailable, selective degrader of BRD9, and that unlike BRD9 inhibition, BRD9 degradation is efficacious in preclinical models of synovial sarcoma."

The company mentioned that the FDA recently approved an investigational new drug (IND) application for CFT8634 in December 2021 allowing C4 Therapeutics to go forward with its Phase 1/2 clinical trial in patients with synovial sarcoma and SMARCB1-null solid tumors. The firm indicated that the study is proceeding and stated that it expects to begin dosing patients in the trial in H1/22.

C4 Therapeutics is based in Watertown, Mass. and is a clinical-stage biopharmaceutical company that is focused on the implementation of targeted protein degradation science used to create a new small-molecule medicines to treat cancer and other diseases with the goal of transforming patients' lives.

C4 Therapeutics Inc. began the day with a market cap of around $1.0 billion with approximately 48.74 million shares outstanding and a short interest of about 6.9%. CCCC shares opened 6% higher today at $22.34 (+$1.34, +6.38%) over yesterday's $21.00 closing price. The stock has traded today between $21.73 and $24.80 per share and is currently trading at $24.28 (+$3.28, +15.62%).


1) Stephen Hytha compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.
2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.
3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.
6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Want to read more about Biotechnology / Pharmaceuticals investment ideas?
Get Our Streetwise Reports Newsletter Free and be the first to know!

A valid email address is required to subscribe