Breaking News FDA Approves OGSIVEO ( nirogacestat)

FDA NEWS RELEASE

FDA Approves First Therapy for Rare Type of Non-Cancerous Tumors

The U.S. Food and Drug Administration approved Ogsiveo (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas.

Desmoid tumors are non-cancerous but can be locally aggressive. The tumors may invade into surrounding structures and organs, resulting in pain, issues with being able to move, and decreased quality of life. Although surgical removal has historically been the treatment of choice, there is a high risk that the tumor will return or that other health challenges will occur after removal; therefore, systemic therapies (cancer treatment targeting the entire body) are being increasingly evaluated in clinical trials. 

“The FDA continues to address unmet medical need and advance the development of safe and effective therapies for the millions of Americans whose lives are affected by rare tumors,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Desmoid tumors are rare tumors that can lead to severe pain and disability. Today’s approval will offer the first approved treatment option for patients beyond surgery and radiation.”

The effectiveness of Ogsiveo was evaluated in an international, multicenter, randomized, double-blind, placebo-controlled trial in 142 adult patients with progressing desmoid tumors not amenable to surgery. Patients were randomized to receive 150 milligrams (mg) of Ogsiveo or placebo orally, twice daily, until disease progression or unacceptable toxicity. The main efficacy outcome measure was progression-free survival (the length of time after the start of treatment for which a person is alive and their cancer does not grow or spread). Objective response rate (a measure of tumor shrinkage) was an additional efficacy outcome measure. 

The pivotal clinical trial demonstrated that Ogsiveo provided clinically meaningful and statistically significant improvement in progression-free survival compared to placebo. Additionally, the objective response rate was also statistically different between the two arms with a response rate of 41% in the Ogsiveo arm and 8% in the placebo arm. The progression-free survival results were also supported by an assessment of patient-reported pain favoring the Ogsiveo arm. 

The most common side effects seen in at least 15% of the patients in the trial were diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection and dyspnea. 

Ogsiveo was granted Priority Review under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition compared to available therapies. Ogsiveo also received FDA Fast Track and Breakthrough Therapy designations for the indication noted above, as well as Orphan-Drug designation for treatment of desmoid tumor (aggressive fibromatosis). Orphan-drug designation provides incentives to assist and encourage drug development for rare diseases.

The FDA granted the approval of Ogsiveo to SpringWorks Therapeutics Inc.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.

Inquiries

Media:
Lauren-Jei McCarthy
240-702-3940

PHASE III SORAFENIB DESMOID TUMOR STUDY

We are thrilled to announce that the recent Phase III Sorafenib Desmoid Tumor Study, has been named by ASCO as one of the top breakthroughs in Oncology in 2018. Named as an “Advance of the Year”, the desmoid study is showcased with others showing “major breakthroughs in clinical cancer research and care.”

The American Society of Clinical Oncology is the world’s leading professional organization of researchers and clinicians who treat people who have cancer. Per ASCO, “rare cancers account for approximately 20% of all cancers diagnosed each year, and incidence rates vary worldwide. Progress has historically lagged behind the achievements made in more common cancers; however, five major studies this past year [including the Sorafenib Desmoid study] offer significant steps forward, making this a notable year for advances in rare cancers.”

We congratulate DTRF Scientific Advisor, Dr. Mrinal Gounder, for his leadership as Principal Investigator of the trial.

Read more in ASCO Special Article: http://ascopubs.org/doi/10.1200/JCO.18.02037

The Sorafenib study has also been highlighted in Conquer Cancer – https://www.conquer.org/advancing-clinical-research

NIH-funded study shows sorafenib improves progression-free survival for patients with rare sarcomas

“Interim results from a randomized clinical trial for patients with desmoid tumors or aggressive fibromatosis (DT/DF) show that the drug sorafenib tosylate (Nexavar) extended progression-free survival compared with a placebo. Progression-free survival is the length of time patients lived before their disease worsened. Based on these interim results, the data and safety monitoring board overseeing the trial recommended that the primary results of the study be released…”
Read full article on the National Institute of Health website.

CLINICAL TRIALS FOR IMMUNOTHERAPY & HIFU

  • Clinical Trial: Phase II DART Immunotherapy “Despite their name, rare cancers make up more than 20 percent of cancers diagnosed worldwide. DART is a clinical trial testing new treatments for dozens of rare cancers. Desmoid tumor is one of the tumor types included in this trial…”
  • Clinical Trial: MR-guided High Intensity Focused Ultrasound (HIFU) for Patients Under 30 Years Old “Children’s National Health System has an open clinical trial using magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU), a noninvasive technology, to destroy solid tumors including desmoid tumors in children, adolescents, and young adults…”

Read more at the Desmoid Tumor Research Foundation website

UPCOMING CLINICAL TRIALS

  • Phase III Trial for Nirogacestat : “A new company named SpringWorks Therapeutics has been formed as a spinoff of Pfizer to focus on investigational therapies that hold significant promise for underserved populations…”
  • Phase I Trial for Tegavivint : “At our 11th DTRF Annual Patient Meeting in Philadelphia, we were pleased to have Beta Cat Pharmaceuticals’ Jon Northrup, Chief Executive Officer, and Casey Cunningham, Chief Medical Officer, present an outline of the planned clinical trial of Tegavivint, a new drug that directly interferes with beta-catenin stabilization…”

Read More at the Desmoid Tumor Research Foundation website

Two Exciting New Research Projects

The Desmoid Tumour Foundation of Canada team would like to thank you for your outstanding contributions from the first ever annual event last May. We are beyond thrilled to have exceeded our goal and are pleased to announce that $70,000 will be allocated to research! Over the past couple of weeks, the medical advisory board met and have decided to fund two new research projects, that are very encouraging to pave the way for a cure.

Duke University – Creation of New Mouse Models

This exciting project, will be creating new mouse models that replicate the specific molecular genetics of the more common types of desmoid tumours. These animals will be made available, not just in Canada, but to researchers throughout the world! By using these genetically advanced mouse models, researchers can work on them to better understand how desmoid tumours grow and to test new drugs and other therapeutic approaches.

Hospital for Sick Kids – Studying the interaction between mutant desmoid cells and normal cells

To learn how to stop a desmoid tumour from growing, means we need to understand how the tumour cells interact with their neighboring normal cells, called stromal cells. In desmoid tumours, it is hard to differentiate the tumour cells from the normal cells because both populations originate from the same family of cells, called mesenchymal cells. After analyzing several desmoid tumour samples, researchers have found an interesting fact; the total tumour mass is actually made up of a high population of NON-mutant cells. It is the INTERACTION between these two types of cells that has researchers intrigued because this interaction somehow controls tumour progression and how a tumour responds to treatment. Disrupting this tumour-normal cell interaction provides new avenues for targeted therapy as is now recognized in other cancers, such as breast and lung cancers. With the help of our donated funds, investigators have created newly developed cell cultures, where they can test different drugs in the hopes of disrupting this cell-cell interaction, which will help identify new treatments.
We are pleased to announce that at the beginning of May we will be hosting our 2nd annual fundraising event!

NEW Facebook Support Group

Hello all Desmoidians! We are pleased to announce that the DTFC has created a closed Facebook Support Group. This group has been created for you and your families. After hearing from a number of Desmoidians, we have learned that you would like to talk amongst other Desmoidians from across Canada, to connect and share your journey and stories. We are encouraging you to join the “Canadian Desmoid Support Group”. Please find us on our Website or Facebook page by clicking on the link.
https://www.facebook.com/groups/2013417335544409/?source_id=1722734144662254

Why Donate

The Desmoid Tumour Foundation of Canada (DFC) is dedicated to funding research for a cure for the rare condition of Desmoid Tumours.

This devastating disease is very rare, affecting approximately 2 in 1 million every year.

Desmoid Tumours, also known as “aggressive fibromatosis,”are locally aggressive and can cause life threatening problems or even death when they invade vital organs.

The tendency for recurrence with surgery makes the treatment of these very rare tumours challenging.

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