Rigel Pharmaceuticals Completes Transfer of GAVRETO® (pralsetinib) New Drug Application
Rigel Pharmaceuticals completed the transfer of the New Drug Application (NDA) for GAVRETO® (pralsetinib), an oral targeted therapy for patients with RET fusion-positive non-small cell lung cancer (NSCLC) and advanced thyroid cancer. GAVRETO will be available in the U.S. starting June 27, 2024. The NSCLC indication is fully approved by the FDA, while the advanced thyroid indication is approved under accelerated approval. The drug will be available in 100 mg capsules priced at $11,144.58 for 60 capsules and $16,716.85 for 90 capsules. Rigel offers patient assistance programs and comprehensive support through its RIGEL ONECARE® service. The acquisition of GAVRETO’s U.S. rights from Blueprint Medicines was announced in February 2024.
- Completion of NDA transfer for GAVRETO.
- GAVRETO available in the U.S. starting June 27, 2024.
- NSCLC indication fully approved by the FDA.
- Addition of GAVRETO to Rigel's commercial portfolio.
- Advanced thyroid indication approved under accelerated approval, pending confirmatory trials.
- Severe side effects include life-threatening ILD/pneumonitis, Grade 3 hypertension, serious hepatic adverse reactions, and fatal hemorrhagic events.
- GAVRETO has a high cost, with bottles priced at $11,144.58 and $16,716.85.
GAVRETO® (pralsetinib) represents a significant treatment option for patients with specific genetic mutations in lung and thyroid cancer. The drug targets RET fusions, which are relatively rare but critical mutations. For non-small cell lung cancer (NSCLC), RET fusions are present in about
The fact that GAVRETO is approved for these indications, albeit with accelerated approval for thyroid cancer, means it has shown promising early results. However, the continued approval for the thyroid cancer indication hinges on ongoing confirmatory trials. Investors should closely monitor the outcomes of these trials as they will determine the long-term viability of this indication.
Another point of interest is the drug's side effect profile, which includes severe potential risks such as interstitial lung disease, hypertension and hepatotoxicity. These adverse effects necessitate careful monitoring and may impact patient adherence and overall market uptake. Nevertheless, GAVRETO’s once-daily oral administration is a convenient option compared to intravenous therapies, which may improve patient compliance.
From a financial perspective, the acquisition of GAVRETO® by Rigel Pharmaceuticals is a strategic move to enhance its oncology portfolio, leveraging its existing distribution and patient support infrastructure. The wholesale acquisition cost (WAC) for GAVRETO is detailed, with prices for different packaging configurations. For instance, a bottle of 90 capsules (100 mg each) costs
However, the potential market size is limited due to the specific genetic mutations GAVRETO targets. NSCLC is common, but RET fusions occur in only a small fraction, approximately
Investors should also consider the competitive landscape. While GAVRETO is presently the only once-daily, oral RET-inhibitor therapy, the market is dynamic and new entrants could emerge, impacting Rigel's market share. The investor should weigh the potential high returns against these risks.
GAVRETO will be available from Rigel in the
"The transfer of
GAVRETO's NSCLC indication is fully approved by the FDA and its advanced thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for the advanced thyroid indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Discussions with the FDA regarding confirmatory requirements are ongoing.
GAVRETO is the only once daily, oral RET-inhibitor therapy that is designed to selectively target RET in mNSCLC and advanced or metastatic thyroid carcinoma. The recommended dosage of GAVRETO is 400 mg taken orally once daily. GAVRETO will be supplied as follows:
NDC Number | Capsule Strength | Package Configuration | WAC Price |
71332-006-60 | 100 mg | Bottles of 60 capsules | |
71332-006-90 | 100 mg | Bottles of 90 capsules |
For those who qualify, Rigel offers patient assistance programs for patients prescribed GAVRETO by their doctor. RIGEL ONECARE®, the company's comprehensive patient support center, can help patients and physicians as they navigate through insurance coverage requirements and provide financial assistance when needed and if eligible, along with other support programs. To learn more, visit www.RIGELONECARE.com or contact RIGEL ONECARE at 833-RIGELOC (833-744-3562).
Rigel announced its acquisition of the
About NSCLC
It is estimated that over 230,000 adults in the
About GAVRETO® (pralsetinib)
INDICATIONS
GAVRETO (pralsetinib) is indicated for the treatment of:
- Adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test
- Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)*
*This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
IMPORTANT SAFETY INFORMATION
- Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, and fatal ILD/pneumonitis can occur in patients treated with GAVRETO. Pneumonitis occurred in
12% of patients who received GAVRETO, including3.3% with Grade 3-4, and0.2% with fatal reactions. Monitor for pulmonary symptoms indicative of ILD/pneumonitis. Withhold GAVRETO and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue GAVRETO based on severity of confirmed ILD. - Hypertension: Occurred in
35% of patients, including Grade 3 hypertension in18% of patients. Overall,8% had their dose interrupted and4.8% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate GAVRETO in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating GAVRETO. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue GAVRETO based on the severity. - Hepatotoxicity: Serious hepatic adverse reactions occurred in
1.5% of patients treated with GAVRETO. Increased aspartate aminotransferase (AST) occurred in49% of patients, including Grade 3 or 4 in7% and increased alanine aminotransferase (ALT) occurred in37% of patients, including Grade 3 or 4 in4.8% . The median time to first onset for increased AST was 15 days (range: 5 days to 2.5 years) and increased ALT was 24 days (range: 7 days to 3.7 years). Monitor AST and ALT prior to initiating GAVRETO, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue GAVRETO based on severity. - Hemorrhagic Events: Serious, including fatal, hemorrhagic events can occur with GAVRETO. Grade ≥3 events occurred in
4.1% of patients treated with GAVRETO including one patient with a fatal hemorrhagic event. Permanently discontinue GAVRETO in patients with severe or life-threatening hemorrhage. - Tumor Lysis Syndrome (TLS): Cases of TLS have been reported in patients with medullary thyroid carcinoma receiving GAVRETO. Patients may be at risk of TLS if they have rapidly growing tumors, a high tumor burden, renal dysfunction, or dehydration. Closely monitor patients at risk, consider appropriate prophylaxis including hydration, and treat as clinically indicated.
- Risk of Impaired Wound Healing: Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, GAVRETO has the potential to adversely affect wound healing. Withhold GAVRETO for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of GAVRETO after resolution of wound healing complications has not been established.
- Embryo-Fetal Toxicity: Based on findings from animal studies and its mechanism of action, GAVRETO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with GAVRETO and for 2 weeks after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with GAVRETO and for 1 week after the last dose.
- Common adverse reactions (≥
25% ) were musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough. Common Grade 3/4 laboratory abnormalities (≥2% ) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased phosphate, decreased leukocytes, decreased sodium, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased calcium (corrected), decreased platelets, increased alkaline phosphatase, increased potassium, decreased potassium, and increased bilirubin. - Avoid coadministration of GAVRETO with strong or moderate CYP3A inhibitors, P-gp inhibitors, or combined P-gp and strong or moderate CYP3A inhibitors. If coadministration cannot be avoided, reduce the GAVRETO dose. Avoid coadministration of GAVRETO with strong or moderate CYP3A inducers. If coadministration cannot be avoided, increase the GAVRETO dose.
- Lactation: Advise women not to breastfeed during treatment with GAVRETO and for 1 week after the last dose.
- Pediatric Use: Monitor open growth plates in adolescent patients. Consider interrupting or discontinuing GAVRETO if abnormalities occur.
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here to see the full Prescribing Information and Patient Information for GAVRETO.
GAVRETO and RIGEL ONECARE are registered trademarks of Rigel Pharmaceuticals, Inc.
About Rigel
Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in
- The American Cancer Society. Key Statistics for Lung Cancer. Revised January 29, 2024. Accessed June 10, 2024: https://www.cancer.org/cancer/types/lung-cancer/about/key-statistics.html
- Kato, S. et al. RET aberrations in diverse cancers: next-generation sequencing of 4,871 patients. Clin Cancer Res. 2017 April 15;23(8):1988-1997. doi: 10.1158/1078-0432.CCR-16-1679
Forward Looking Statements
This press release contains forward-looking statements relating to, among other things, the potential benefits of Rigel's acquisition of
Contact for Investors & Media:
Investors:
Rigel Pharmaceuticals, Inc.
650.624.1232
ir@rigel.com
Media:
David Rosen
Argot Partners
212.600.1902
david.rosen@argotpartners.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/rigel-pharmaceuticals-completes-transfer-of-gavreto-pralsetinib-new-drug-application-302180754.html
SOURCE Rigel Pharmaceuticals, Inc.
FAQ
When will GAVRETO be available in the U.S.?
What is the price of GAVRETO?
What is GAVRETO used for?
Is GAVRETO fully approved by the FDA?