Marstacimab

Pfizer Discontinues Beqvez Gene Therapy for Hemophilia B Amid Low Market Demand

3 months ago

• Pfizer has announced the cessation of development and commercialization of Beqvez, its FDA-approved gene therapy for hemophilia B, citing limited interest from patients and healthcare providers. • The decision marks Pfizer's complete exit from viral gene replacement therapies, following earlier withdrawals from Duchenne muscular dystrophy and hemophilia A programs. • The company will shift focus to other treatments including Hympavzi for hemophilia A, while maintaining support for existing Beqvez clinical trial participants.

Fitusiran Shows Promise in Reducing Bleeding Episodes in Hemophilia A and B Patients

4 months ago

• Fitusiran, an RNA interference therapy, is highlighted as a potential breakthrough for hemophilia A and B treatment, regardless of inhibitor status. • Phase 3 trials (NCT03417245) demonstrated that fitusiran significantly reduced annualized bleeding rates in hemophilia A and B patients compared to on-demand factor concentrates. • Another study (NCT03417102) showed a 90.8% reduction in annualized bleeding rate in patients with inhibitors treated with fitusiran compared to on-demand treatment. • Fitusiran's efficacy and convenient subcutaneous administration position it as a drug to watch in hematology, offering a more personalized approach to hemophilia management.

Hemgenix Gene Therapy Shows Sustained Efficacy in Hemophilia B Patients After Four Years

4 months ago

• Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B. • Patients treated with Hemgenix experienced a 90% reduction in adjusted annualized bleeding rate (ABR) at year four compared to the lead-in period. • 94% of patients remained free of continuous prophylaxis treatment at year four, indicating sustained independence from regular infusions. • The gene therapy maintained a favorable safety profile, with most adverse events occurring within the first six months post-treatment.

Pfizer's Gene Therapy Fidanacogene Elaparvovec Shows Promise in Haemophilia B Treatment

5 months ago

• Pfizer's fidanacogene elaparvovec (SPK-9001) gene therapy met its primary endpoint in the Phase 3 BENEGENE-2 study for haemophilia B, demonstrating non-inferiority and superiority in annualized bleeding rate. • The BENEGENE-2 study showed a 71% reduction in annualized bleeding rate (ABR) with fidanacogene elaparvovec compared to the pre-treatment period in adult males with moderately severe to severe haemophilia B. • Secondary endpoints revealed a 78% reduction in treated ABR and a 92% reduction in annualized infusion rate, with a safety profile consistent with earlier phase studies. • Fidanacogene elaparvovec has received breakthrough therapy designations from regulatory agencies, potentially offering a one-time treatment option to reduce the clinical burden for haemophilia B patients.

Capricor Therapeutics Completes FDA Submission for Deramiocel in DMD Cardiomyopathy

5 months ago

• Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. • The BLA is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, showing attenuation of cardiac implications of DMD. • The FDA has been requested to grant priority review, potentially reducing the review period to six months from the standard ten months. • The BLA submission triggers a $10 million milestone payment to Capricor from its distribution partner, Nippon Shinyaku.

FDA Approves New Therapies for Hemophilia, Colorectal Cancer, and Cystic Fibrosis

5 months ago

• The FDA approved Alhemo (concizumab) from Novo Nordisk for hemophilia A and B with inhibitors, offering a once-daily subcutaneous option. • Pfizer's Braftovi (encorafenib) received accelerated approval as a first-line treatment for BRAF V600E-mutated metastatic colorectal cancer in combination with other therapies. • Vertex Pharma's Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a new triplet therapy for cystic fibrosis, has been approved for patients aged six and older with responsive mutations. • Hikma Pharma gains FDA approval for the first generic version of Novo Nordisk's Victoza (liraglutide) for treating type 2 diabetes.

FDA Approves Novo Nordisk's Alhemo for Hemophilia A and B with Inhibitors

5 months ago

• The FDA has approved Alhemo (concizumab-mtci) as a once-daily prophylactic treatment for hemophilia A and B with inhibitors in patients 12 years and older. • Alhemo, a subcutaneous injection, is the first of its kind for this patient population, offering an alternative to intravenous infusions. • Clinical trials showed an 86% reduction in treated bleeding episodes with Alhemo compared to no prophylaxis, significantly improving patient outcomes. • Alhemo works by blocking tissue factor pathway inhibitor (TFPI), enhancing thrombin production to facilitate blood clotting in the presence of inhibitors.

Hemophilia Treatment Advances: Gene Therapy, Prophylaxis, and On-Demand Therapies Highlighted at ASH 2024

5 months ago

• ASH 2024 highlighted significant progress in hemophilia care since Hemlibra's approval, including gene therapies for hemophilia A and B, offering curative potential. • Despite advances, unmet needs persist, including spontaneous bleeds in prophylaxis patients, limited gene therapy access, and concerns about long-term risks like liver toxicity. • Emerging therapies like Pfizer's giroctocogene fitelparvovec and Sanofi's fitusiran show promise, with ongoing trials and regulatory reviews potentially expanding treatment options. • Novel on-demand treatments like Staidson's bemiltenase alfa offer alternatives, particularly in price-sensitive markets, demonstrating a 12-hour bleed clearance rate of 83%.

FDA Wraps Up 2024 with Key Approvals for Drugs Targeting Various Conditions

6 months ago

• The FDA approved Vertex's Alyftrek for cystic fibrosis, offering improved dosing and potential market exclusivity. • Novo Nordisk's Alhemo was approved for hemophilia A and B, providing a new option for patients with inhibitors. • Bristol Myers Squibb's Opdivo Qvantig gained approval as a subcutaneous formulation, offering faster administration for various solid tumors. • Eli Lilly's Zepbound secured approval for obstructive sleep apnea in obese adults, marking the first prescription medicine for this condition.

Pfizer's Hympavzi Receives EU Approval, Novartis Raises Sales Outlook

6 months ago

• The European Commission has approved Pfizer's Hympavzi (marstacimab) for hemophilia A and B treatment, based on positive Phase III BASIS study data. • Novartis has increased its mid-term sales growth guidance, projecting a 5% CAGR for 2024-2029, driven by key drugs and pipeline candidates. • Lilly's muvalaplin demonstrated significant reduction in lipoprotein(a) levels in a Phase II study, showing promise for cardiovascular risk reduction.

European Commission Approves Pfizer's Hympavzi for Haemophilia A and B

6 months ago

• The European Commission (EC) has approved Pfizer's Hympavzi (marstacimab) for routine prophylaxis of bleeding episodes in haemophilia A and B patients. • Hympavzi is the first anti-tissue factor pathway inhibitor approved in the EU for haemophilia A or B, administered via a pre-filled, auto-injector pen. • The approval was supported by the late-stage BASIS trial, which demonstrated a 35% reduction in annualized bleeding rate compared to factor VIII or IX prophylaxis. • Hympavzi is administered as a once-weekly subcutaneous injection, reducing the amount of tissue factor pathway inhibitor and increasing thrombin generation.

EU Approves Pfizer's Hympavzi for Hemophilia A and B

6 months ago

• The European Commission has granted marketing authorization for Pfizer's Hympavzi (marstacimab) for hemophilia A and B patients aged 12 and older. • Hympavzi is a once-weekly prophylactic treatment to reduce bleeding episodes in patients without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). • The approval is based on Phase III BASIS study data, demonstrating a statistically significant reduction in annualized bleeding rate (ABR) compared to standard care. • Hympavzi targets tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein, and is already approved in the United States.

EU Approves Pfizer's Hympavzi for Severe Hemophilia A and B

6 months ago

• The European Commission has granted marketing authorization for Hympavzi, a new treatment developed by Pfizer. • Hympavzi is indicated for severe hemophilia A or B in both adult and adolescent patients without inhibitors. • This approval marks a significant advancement in the treatment landscape for individuals living with severe hemophilia. • Hympavzi offers a novel therapeutic option for patients who have limited treatment choices.

European Commission Approves Pfizer's HYMPAVZI for Hemophilia A and B

6 months ago

• The European Commission has granted marketing authorization to Pfizer's HYMPAVZI (marstacimab) for treating hemophilia A and B based on Phase 3 trial results. • HYMPAVZI is the first once-weekly, subcutaneous treatment in the EU for severe hemophilia B and can be administered via a pre-filled, auto-injector pen. • Clinical trials demonstrated HYMPAVZI significantly reduced the annual bleeding rate by 35% compared to routine prophylaxis with FVIII or FIX. • This approval marks a significant advancement, offering a convenient treatment option that reduces the burden of frequent intravenous infusions for hemophilia patients.

EU Approves Pfizer's Hympavzi as First Weekly Subcutaneous Treatment for Severe Hemophilia B

6 months ago

• The European Commission has approved Pfizer's Hympavzi, a once-weekly subcutaneous treatment, for severe hemophilia B in adults and adolescents without inhibitors. • Hympavzi is the first anti-TFPI antibody approved in Europe for hemophilia A or B without inhibitors, offering convenient at-home administration via autoinjector. • The approval was based on the Phase 3 BASIS study, which demonstrated a 35% reduction in annualized bleeding rate compared to routine clotting factor injections. • Hympavzi provides a new treatment option with simplified, weight-independent dosing, potentially reducing the treatment burden for hemophilia B patients.

European Commission Approves Pfizer's Hympavzi for Hemophilia A and B

6 months ago

• The European Commission has approved Pfizer's Hympavzi for routine prevention of bleeding episodes in hemophilia A and B patients aged 12 and older. • Hympavzi is the first once-weekly subcutaneous treatment in the EU for severe hemophilia B, offering a new administration route. • The approval extends to patients with severe hemophilia A, providing the first pre-filled pen or syringe option in the EU. • The U.S. Food and Drug Administration (FDA) had previously approved Hympavzi in October, marking its global expansion.

FDA Approves Hympavzi (marstacimab-hncq) as Novel Treatment for Hemophilia A and B

6 months ago

• The FDA has approved Hympavzi (marstacimab-hncq), a novel monoclonal antibody, for hemophilia A and B in patients 12 years and older. • Hympavzi offers a new mechanism by reducing tissue factor pathway inhibitor, enhancing thrombin production to facilitate blood clotting. • Unlike traditional therapies requiring frequent administration, Hympavzi is administered once weekly, improving patient convenience. • Clinical trial data showed bleeding rates with Hympavzi were comparable to prophylactic factor replacement in hemophilia A and B patients.

FDA Approves Pfizer's Hympavzi (marstacimab-hncq) for Hemophilia A and B

7 months ago

• The FDA has approved Hympavzi (marstacimab-hncq) for routine prophylaxis to reduce bleeding episodes in hemophilia A and B patients without inhibitors, aged 12 and older. • Hympavzi is the first anti-TFPI therapy approved in the U.S. for hemophilia A or B, offering a once-weekly subcutaneous administration via a pre-filled auto-injector pen. • Clinical trials showed Hympavzi reduced annualized bleeding rates by 35% compared to routine prophylaxis and 92% compared to on-demand treatment in hemophilia A and B patients. • Hympavzi represents a significant advancement, potentially reducing the treatment burden for patients who require frequent intravenous infusions, improving their quality of life.

FDA Approves Hympavzi (marstacimab-hncq) for Hemophilia A and B Treatment

7 months ago

• The FDA has approved Hympavzi (marstacimab-hncq) for routine prophylaxis to reduce bleeding episodes in hemophilia A without factor VIII inhibitors and hemophilia B without factor IX inhibitors. • Hympavzi is a novel treatment that targets tissue factor pathway inhibitor, an anticoagulation protein, to enhance thrombin generation and improve blood clotting. • Clinical trial results showed Hympavzi was superior to on-demand factor replacement and provided similar bleeding rates to prophylactic factor replacement in hemophilia patients. • The approval marks a significant advancement, offering a new therapeutic option for both adult and pediatric patients (12 years and older) with hemophilia A and B.

FDA Gears Up for Busy November with Key Decisions on Novel Therapies

7 months ago

• The FDA is set to decide on Journey Medical's DFD-29 for rosacea, potentially the first oral systemic therapy addressing both inflammatory lesions and erythema. • Merus' Zenocutuzumab, a targeted therapy for NRG1+ lung and pancreatic cancers, is under priority review, offering a potential improvement over current treatments. • Decisions are pending for PTC Therapeutics' Upstaza for AADC deficiency, Autolus Therapeutics' Obe-cel for B-cell acute lymphoblastic leukemia, and Applied Therapeutics' Govorestat for Classic Galactosemia.

Drug Development Updates