Pertuzumab
- Generic Name
- Pertuzumab
- Brand Names
- Perjeta, Perjeta-Herceptin, Phesgo
- Drug Type
- Biotech
- CAS Number
- 380610-27-5
- Unique Ingredient Identifier
- K16AIQ8CTM
- Background
Pertuzumab is a recombinant humanized monoclonal antibody that targets the extracellular dimerization domain (subdomain II) of the human epidermal growth factor receptor 2 protein (HER2). It consists of two heavy chains and two lights chains that have 448 and 214 residues respectively. It was first approved by the FDA in 2012 for use with docetaxel and another HER2-targeted monoclonal antibody, trastuzumab, in the treatment of metastatic HER2-positive breast cancer. Its indicated conditions have since expanded to include use as both a neoadjuvant therapy and an adjuvant therapy in the treatment of HER2-positive breast cancers at high risk of recurrence.
- Indication
Pertuzumab is indicated for intravenous administration in combination with trastuzumab and docetaxel for the treatment of patients with HER2-positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease. It is also indicated in combination with trastuzumab and other chemotherapies for the neoadjuvant treatment of HER2-positive locally advanced, inflammatory, or early-stage breast cancer as part of a complete treatment regimen and as adjuvant treatment in patients with HER2-positive early-stage breast cancer at high risk of recurrence.
Pertuzumab is also indicated for subcutaneous injection - in combination with trastuzumab and hyaluronidase - in the treatment of HER2-positive breast cancers in adults.
- Associated Conditions
- Inflammatory Breast Cancer (IBC), Locally Advanced Breast Cancer (LABC), Metastatic Breast Cancer, Stage I Breast Cancer
Ten-Year APHINITY Data Shows Perjeta-Based Regimen Reduces Death Risk by 17% in HER2-Positive Early Breast Cancer
• Long-term follow-up data from the Phase III APHINITY trial demonstrates a statistically significant 17% reduction in risk of death when adding Perjeta (pertuzumab) to Herceptin (trastuzumab) and chemotherapy in early-stage HER2-positive breast cancer.
• The benefit was more pronounced in patients with lymph node-positive disease, showing a 21% reduction in death risk, reinforcing the regimen's value as a standard-of-care treatment in the curative setting.
• After ten years, 91.6% of patients receiving the Perjeta-based regimen were alive compared to 89.8% in the control group, with the previously reported invasive disease-free survival benefit maintained without new safety concerns.
Roche and Regeneron Announce Multi-Billion Dollar Investments in US Manufacturing Amid Tariff Concerns
• Roche has committed $50 billion to expand its US operations over five years, including new manufacturing facilities and R&D centers, with plans to create over 12,000 new jobs nationwide.
• Regeneron is investing more than $3 billion in US operations through a partnership with Fujifilm Diosynth Biotechnologies, nearly doubling its large-scale manufacturing capacity in the country.
• These pharmaceutical investments come as President Trump continues to threaten sector-specific tariffs, with Roche stating it will eventually export more medicines from the US than it imports.
Roche's OCREVUS Shows Strong Efficacy in MUSETTE Phase III Trial for Multiple Sclerosis
• Roche Holding's MUSETTE Phase III trial demonstrated strong efficacy of the approved 600 mg dose of OCREVUS for multiple sclerosis, reinforcing its position in the treatment landscape.
• The company has launched a new subcutaneous formulation of OCREVUS, expanding administration options for patients while continuing to build its neuromuscular disease portfolio.
• Roche has outperformed both the Swiss market and pharmaceutical industry with a 33.01% total shareholder return over the past year, bolstered by strategic collaborations and regulatory approvals.
Novel BCMA-Directed CAR T-Cell Therapy Shows Promising Efficacy in Relapsed/Refractory Multiple Myeloma and AL Amyloidosis
• A second-generation BCMA-directed CAR T-cell therapy, MDC-CAR-BCMA001, demonstrated remarkable efficacy in patients with relapsed/refractory multiple myeloma and AL amyloidosis, achieving a 5/6 overall response rate with 4 complete responses.
• The novel therapy showed a favorable safety profile with manageable toxicity, including low incidence of severe cytokine release syndrome and no neurotoxicity, even in patients with significant organ dysfunction.
• These promising results have prompted further investigation through ongoing clinical trials, including CARLOTTA001 (NCT05836896) and the CLEAR AL trial, potentially offering new hope for difficult-to-treat hematologic malignancies.
U.S. Monoclonal Antibodies Market Projected to Reach $284.9 Billion by 2034, Growing at 13.1% CAGR
• The U.S. monoclonal antibodies market is expected to grow from $94.09 billion in 2025 to $284.90 billion by 2034, driven by rising prevalence of chronic diseases and expanding therapeutic applications beyond oncology.
• Human-source monoclonal antibodies dominated the market with 34.3% share in 2024, while oncology applications led with 38.2% of market value, reflecting the critical role of mAbs in targeted cancer therapies.
• Artificial intelligence integration is revolutionizing the mAb industry by accelerating drug discovery, optimizing manufacturing processes, and enabling more personalized treatments with improved efficacy and reduced development costs.
Bluejay Therapeutics Strengthens Leadership Team with Appointment of Veteran Biotech Executive Mary Cromwell
• Bluejay Therapeutics has appointed Mary Cromwell, Ph.D., as Senior Vice President and Head of Technical Operations, bringing over 30 years of biotechnology operations experience to advance their liver disease therapeutics pipeline.
• Dr. Cromwell joins from Allakos where she served as Chief Technology Officer, following an extensive career at Genentech/Roche where she contributed to the licensure of major products including Avastin® and Perjeta®.
• The appointment comes as Bluejay's lead candidate brelovitug (BJT-778) advances in development for chronic hepatitis D, having recently received FDA Breakthrough Therapy designation and EMA's PRIME and Orphan designation.
FDA Reviews Pertuzumab Biosimilar HLX11 for HER2-Positive Breast Cancer Treatment
• Shanghai Henlius Biotech's pertuzumab biosimilar HLX11 has received FDA acceptance of its Biologics License Application for HER2-positive breast cancer treatment, supported by comprehensive clinical trials.
• Phase 1 study demonstrated comparable pharmacokinetics, safety, and immunogenicity between HLX11 and reference pertuzumab across US, EU, and Chinese versions of the drug.
• Phase 3 trial successfully met its primary endpoint of total pathological complete response in HER2-positive, hormone receptor-negative early or locally advanced breast cancer patients.
Datopotamab Deruxtecan's Role in HR+/HER2- Breast Cancer Treatment: Sequencing and Safety Considerations
• Datopotamab deruxtecan (Dato-DXd) is poised to become a standard treatment for metastatic hormone receptor-positive, HER2-negative breast cancer after prior systemic therapy.
• Optimal sequencing of antibody-drug conjugates (ADCs) like Dato-DXd, sacituzumab govitecan, and trastuzumab deruxtecan remains uncertain, especially considering overlapping toxicities.
• Real-world toxicity profiles suggest sacituzumab govitecan may cause more cytopenias and diarrhea, while Dato-DXd and trastuzumab deruxtecan are linked to interstitial lung disease.
• The lack of overall survival difference in the TROPION-Breast01 trial raises questions about efficacy issues or the impact of crossover between treatment arms.
KN026 Plus Docetaxel Shows Promise in HER2+ Metastatic Breast Cancer
• A Phase 2 trial of KN026 plus docetaxel shows a 76.4% objective response rate in HER2+ metastatic breast cancer patients.
• The combination therapy resulted in a median progression-free survival of 27.7 months, indicating a potential first-line treatment option.
• The study reports a manageable safety profile, with no deaths attributable to KN026 or docetaxel, suggesting a favorable cardiac safety profile.
• A Phase 3 trial is planned to further evaluate the efficacy and safety of KN026 in combination with HB1801 for HER2+ metastatic breast cancer.
Kadcyla Shows Sustained Survival Benefit in HER2-Positive Early Breast Cancer
• Long-term analysis of the KATHERINE trial reveals that Kadcyla (T-DM1) significantly improves overall survival in HER2-positive early breast cancer patients with residual invasive disease.
• The study demonstrated a 7-year overall survival rate of 89.1% with T-DM1 compared to 84.4% with trastuzumab monotherapy, indicating a substantial survival advantage.
• Kadcyla also showed a sustained improvement in invasive disease-free survival, with a 7-year rate of 80.8% versus 67.1% for trastuzumab, reinforcing its efficacy.
• The safety profile of T-DM1 was acceptable, with manageable adverse events, further supporting its use in the adjuvant treatment of high-risk early breast cancer.
T-DM1 Shows Sustained Survival Benefit in HER2-Positive Breast Cancer
• Adjuvant trastuzumab emtansine (T-DM1) demonstrates significantly improved overall survival compared to trastuzumab in HER2-positive early breast cancer patients with residual invasive disease.
• The KATHERINE trial's long-term follow-up reveals a sustained benefit in invasive disease-free survival with T-DM1, reducing the risk of recurrence or death.
• T-DM1's consistent benefit across patient subgroups, regardless of disease extent or hormone receptor status, reinforces its role as a standard of care.
• While adverse events were more frequent with T-DM1, the overall safety profile remains acceptable, supporting its use in high-risk HER2-positive breast cancer.
Novel HER2-Targeted Therapies Show Promise in Overcoming Treatment Resistance in Advanced Breast Cancer
• New HER2-directed antibody-drug conjugate SHR-A1811 demonstrates impressive 76.3% response rate in HER2-positive breast cancer patients, with potentially improved safety profile compared to existing treatments.
• Bispecific antibody zanidatamab combined with evorpacept shows 55.6% response rate in heavily pretreated patients who progressed on T-DXd, offering hope for resistant disease.
• Research reveals potential resistance mechanisms to HER2-targeted therapies, including topoisomerase I alterations and HER2 gene loss, helping guide future treatment strategies.
FDA Approves Seattle Genetics' Advanced Breast Cancer Drug Tukysa
The FDA has granted early approval to Seattle Genetics' breast cancer drug Tukysa (tucatinib) for advanced HER2-positive breast cancer, including cases with brain metastases, marking the company's first approval in breast cancer treatment.
Enhertu Approved for HER2-Low and HER2-Ultralow Metastatic Breast Cancer
• The FDA has approved Enhertu for HR-positive, HER2-low or HER2-ultralow metastatic breast cancer after endocrine therapy progression.
• DESTINY-Breast06 trial data showed a 36% reduction in disease progression or death risk compared to chemotherapy.
• Patients on Enhertu had a median progression-free survival of 13.2 months versus 8.1 months on chemotherapy.
• This approval expands Enhertu's use to an earlier treatment setting and a broader patient population.
Kymriah Receives EU Approval as First CAR-T Therapy for Follicular Lymphoma
• Novartis' Kymriah is the first CAR-T therapy approved in the EU for relapsed/refractory follicular lymphoma, offering a new option for patients after multiple treatments.
• The approval was based on the ELARA trial, demonstrating an 86% response rate and 69% complete response rate in patients treated with Kymriah.
• Kymriah provides durable treatment effects, with 87% of complete responders remaining in remission for at least nine months after initial response.
• This approval expands Kymriah's indications in the EU, adding to its existing approvals for diffuse large B-cell lymphoma and acute lymphoblastic leukemia.
Imlunestrant Shows Promise in Advanced ER+/HER2- Breast Cancer Treatment
• Imlunestrant monotherapy significantly improved progression-free survival (PFS) in advanced breast cancer patients with ESR1 mutations compared to standard endocrine therapy.
• The combination of imlunestrant and abemaciclib demonstrated a statistically significant improvement in PFS compared to imlunestrant alone, regardless of ESR1 mutation status.
• The EMBER-3 trial results suggest imlunestrant, particularly in combination with abemaciclib, could offer a new all-oral targeted therapy option for pre-treated advanced breast cancer.
• Safety data from the trial indicated that imlunestrant, both as a monotherapy and in combination, was generally well-tolerated, with manageable adverse events.
DATROWAY® (Datopotamab Deruxtecan) Receives EU Approval for Previously Treated Metastatic HR+/HER2- Breast Cancer
• DATROWAY, a TROP2-directed antibody drug conjugate (ADC), has been approved in the European Union for treating adult patients with unresectable or metastatic HR+/HER2- breast cancer who have received endocrine therapy and at least one line of chemotherapy.
• The approval is based on the TROPION-Breast01 phase 3 trial, which showed DATROWAY reduced the risk of disease progression or death by 37% compared to chemotherapy, with a median PFS of 6.9 months versus 4.9 months.
• This marks the second ADC approved for breast cancer based on Daiichi Sankyo's DXd technology and the third medicine from their oncology pipeline to receive EU approval, highlighting their commitment to developing innovative cancer treatments.
FDA Approves Ziihera (zanidatamab-hrii) for HER2-Positive Biliary Tract Cancer
• The FDA granted accelerated approval to Ziihera (zanidatamab-hrii) for adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer (BTC).
• Approval was based on a 52% objective response rate and a median duration of response of 14.9 months in the HERIZON-BTC-01 trial.
• Ziihera is the first dual HER2-targeted bispecific antibody and chemotherapy-free treatment option for patients with HER2-positive BTC.
• A confirmatory Phase 3 trial is ongoing to evaluate zanidatamab in combination with standard-of-care therapy versus standard-of-care therapy alone.
Novel HER2-Targeted Therapies Show Promise in Advanced Breast Cancer
• Trastuzumab duocarmazine significantly improves progression-free survival compared to physician's choice in pretreated HER2-positive metastatic breast cancer patients.
• Combining eribulin with trastuzumab and pertuzumab demonstrates non-inferior progression-free survival versus taxanes in first-line HER2-positive advanced breast cancer.
• Pertuzumab retreatment with trastuzumab and chemotherapy shows a significant overall survival benefit in HER2-positive locally advanced/metastatic breast cancer.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
Drug Development Updates
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