The U.S. Food and Drug Administration (FDA) has approved REVTORPYK™ (gedatolisib) for certain people with hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer whose tumors do not have a PIK3CA mutation and whose cancer has progressed after endocrine (hormone) therapy. Here’s what this new approval means and who may benefit.
What is HR-positive, HER2-negative breast cancer?
Not all breast cancers look and act the same. Some grow more quickly than others, and some respond better to certain treatments. Doctors classify breast cancers based on certain proteins found on cancer cells to help determine which treatments are most likely to be effective.
Approximately 70% of breast cancers are hormone receptor (HR)-positive and HER2-negative, making this the most common subtype of breast cancer. Many people with HR-positive breast cancer respond well to hormone-blocking medications. However, over time, some cancers become resistant to these treatments and continue growing. When that occurs, additional treatment options are needed.
Why does PIK3CA status matter?
Doctors often also test advanced breast cancers for changes (mutations) in a gene called PIK3CA. Some treatments are designed specifically for cancers with a PIK3CA mutation. However, about 60% of HR-positive, HER2-negative advanced breast cancers do not carry this mutation, meaning many patients without it have had fewer targeted treatment options available.
The new approval of REVTORPYK specifically addresses this group of patients.
How does REVTORPYK work?
Cancer cells often rely on internal communication networks that tell them to grow and survive. One of the most important of these is called the PI3K/AKT/mTOR pathway.
REVTORPYK is designed to block several key parts of this pathway at the same time. By shutting down multiple signals that cancer cells use, the medication may slow or stop tumor growth more effectively than treatments that target only one part of the pathway.
Rather than being used alone, REVTORPYK is given together with other standard breast cancer treatments: Fulvestrant, or Fulvestrant plus palbociclib, depending on an individual’s treatment plan
What did the clinical trial show?
The FDA approval was based on results from the international Phase 3 VIKTORIA-1 clinical trial, which included patients whose HR-positive, HER2-negative advanced breast cancer had progressed after previous hormone therapy and CDK4/6 inhibitor treatment.
The study showed that REVTORPYK helped keep patients’ cancer under control for longer than standard treatment alone. Patients who received REVTORPYK plus palbociclib and fulvestrant had a 76% lower risk of their cancer progressing or of death compared with patients who received fulvestrant alone. Those who received REVTORPYK plus fulvestrant had a 67% lower risk of disease progression or death. In addition, tumors were more likely to shrink in patients receiving REVTORPYK, an encouraging sign that the treatment was helping to slow the disease.
While these results are encouraging, researchers will continue following patients to better understand long-term outcomes.
Who may be eligible?
REVTORPYK is approved for adults who have:
- HR-positive, HER2-negative locally advanced or metastatic breast cancer
- No detectable PIK3CA mutation
- Disease that has progressed after at least one line of endocrine therapy in the metastatic setting
Your oncologist can determine whether genetic testing and your treatment history make you a candidate for this therapy.
What side effects should patients know about?
Like all cancer treatments, REVTORPYK can cause side effects. Many are manageable with close monitoring and supportive care.
Some of the more common or important side effects include:
- Mouth sores (stomatitis)
- Rash
- Elevated blood sugar (hyperglycemia)
- Fatigue
- Nausea
- Vomiting
- Diarrhea
- Changes in blood cell counts
- Elevated liver enzymes
Because mouth sores were common during clinical trials, patients are typically advised to begin using a steroid-containing, alcohol-free mouthwash before starting treatment to help reduce this risk. Your healthcare team will also monitor blood sugar levels, blood counts, liver function, and other laboratory tests throughout treatment. Patients who are pregnant or planning pregnancy should discuss treatment risks with their healthcare provider.
What does this approval mean?
The approval of REVTORPYK represents an important step forward for patients with HR-positive, HER2-negative metastatic breast cancer who do not have a PIK3CA mutation, as this group has had relatively limited targeted treatment options. By targeting one of the key growth pathways that cancer cells depend on, this new therapy offers another option for slowing disease progression after hormone therapy is no longer effective.
Although REVTORPYK is not a cure for metastatic breast cancer, it expands the growing list of personalized treatments available and highlights the continued progress being made in precision oncology.
Questions to Ask Your Doctor:
- Is my breast cancer HR-positive and HER2-negative?
- Has my tumor been tested for a PIK3CA mutation?
- Am I eligible for REVTORPYK?
- How is this treatment given?
- What side effects should I expect, and how can they be managed?
- How will we know if the treatment is working?
Source
Celcuity. FDA Approves REVTORPYK™ (gedatolisib) for HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer. News release. July 14, 2026.
