- |||||||||| Juxtapid (lomitapide) / Novelion, Chiesi
Journal: Lomitapide: A Medication Use Evaluation and a Formulary Perspective. (Pubmed Central) - May 10, 2024
This review revealed important considerations when reimbursing expensive medications for rare diseases. Real-world evidence in real-time can support healthcare systems in price negotiations and reaching mutual agreements that can eventually improve patient access to care.
- |||||||||| Evkeeza (evinacumab-dgnb) / Regeneron
Journal: Evinacumab and Cardiovascular Outcome in Patients With Homozygous Familial Hypercholesterolemia. (Pubmed Central) - May 2, 2024
No patients on evinacumab experienced cardiovascular events versus 13 events for 5/21 (24%) over 4 years in the control cohort (likelihood P=0.0267). Real-life, long-term evinacumab adjunctive to lipid-lowering therapy including lipoprotein apheresis led to sustained low-density lipoprotein cholesterol lowering and improved cardiovascular event-free survival of patients with HoFH.
- |||||||||| Juxtapid (lomitapide) / Novelion, Chiesi
Journal: Efficient Delivery of Lomitapide Using Hybrid Membrane-Coated Tetrahedral DNA Nanostructures for Glioblastoma Therapy. (Pubmed Central) - Apr 3, 2024
The results showed that the LMP RFA NPs exhibited superior and specific anti-GBM activities, reduced off-target drug delivery, prolonged lifespan and had negligible side effects in tumor-bearing mice. This study combines high-throughput drug screening with biomimetic nanodrug delivery system technology to provide a theoretical and practical basis for drug development and the optimization of clinical treatment strategies for GBM treatment.
- |||||||||| Journal: Dyslipidemia - the known unknown. (Pubmed Central) - Nov 3, 2023
As of April 2023, the portfolio of lipid-lowering medication has been expanded to include inclisiran (small interfering RNA against proprotein convertase subtilisin/kexin type 9 (PCSK9)), which is administered directly in cardiologists' and internists' outpatient clinics, ensuring 100% adherence. In severe hypertriglyceridaemia, fibrate monotherapy may be indicated in addition to dietary and regimen measures; if this treatment fails, some patients may be offered lomitapide, volanesorsen or evinacumab as part of clinical trials or specific treatment programmes if very strict indication criteria are met.
- |||||||||| Evkeeza (evinacumab-dgnb) / Regeneron, Ultragenyx
Journal: Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia. (Pubmed Central) - Oct 20, 2023
P3
In severe hypertriglyceridaemia, fibrate monotherapy may be indicated in addition to dietary and regimen measures; if this treatment fails, some patients may be offered lomitapide, volanesorsen or evinacumab as part of clinical trials or specific treatment programmes if very strict indication criteria are met. Evinacumab constitutes a new treatment for pediatric patients with HoFH and inadequately controlled LDL-C despite optimized lipid-lowering therapy, lowering LDL-C levels by nearly half in these extremely high-risk and difficult-to-treat individuals.
- |||||||||| Praluent (alirocumab) / Sanofi, Regeneron, Juxtapid (lomitapide) / Novelion, Chiesi, Nexletol (bempedoic acid) / Esperion Therap, Otsuka, Daiichi Sankyo
Review, Journal: Advances in Treatment of Dyslipidemia. (Pubmed Central) - Sep 13, 2023
Our review demonstrates the pivotal roles played by each of these drugs in targeting specific parameters of lipid metabolism. We outline the future landscape of dyslipidemia treatment, envisaging a more tailored and effective therapeutic approach to address this widespread medical concern.
- |||||||||| Journal: Established and Emerging Lipid-Lowering Drugs for Primary and Secondary Cardiovascular Prevention. (Pubmed Central) - Jul 24, 2023
Established pharmaceutical treatment options include the Niemann-Pick-C1 like-1 protein (NPC1L1) inhibitor ezetimibe, the protein convertase subtilisin-kexin type 9 (PCSK9) inhibitors alirocumab and evolocumab, fibrates as peroxisome proliferator receptor alpha (PPAR-?) activators, and the omega-3 fatty acid icosapent ethyl...For secondary prevention in hypertriglyceridemia, second-line options such as statin add-on or statin-intolerant treatments are icosapent ethyl and fenofibrate...Recent biotechnological advances have led to the development of innovative small molecules (bempedoic acid, lomitapide, pemafibrate, docosapentaenoic and eicosapentaenoic acid), antibodies (evinacumab), antisense oligonucleotides (mipomersen, volanesorsen, pelcarsen, olezarsen), small interfering RNA (inclisiran, olpasiran), and gene therapies for patients with dyslipidemia...Apart from statins, data on new drugs' use in primary cardiovascular prevention remain scarce. For their swift adoption into clinical routine, these treatments must demonstrate safety and efficacy as well as cost-effectiveness in randomized cardiovascular outcome trials.
- |||||||||| Juxtapid (lomitapide) / Novelion, Chiesi, Evkeeza (evinacumab-dgnb) / Regeneron, Ultragenyx
Journal: 2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance. (Pubmed Central) - Jul 3, 2023
To improve HoFH care around the world, the statement recommends the creation of national screening programmes, education to improve awareness, and management guidelines that account for the local realities of care, including access to specialist centres, treatments, and cost. This updated statement provides guidance that is crucial to early diagnosis, better care, and improved cardiovascular health for patients with HoFH worldwide.
- |||||||||| Juxtapid (lomitapide) / Novelion, Chiesi
Preclinical, Journal: The Beneficial Effect of Lomitapide on the Cardiovascular System in LDLr Mice with Obesity. (Pubmed Central) - Jun 28, 2023
This updated statement provides guidance that is crucial to early diagnosis, better care, and improved cardiovascular health for patients with HoFH worldwide. Treatment with lomitapide improves cardiovascular function and lipid profile and reduces body weight and inflammatory markers in LDLr/ mice on HFD.
- |||||||||| Evkeeza (evinacumab-dgnb) / Regeneron, Ultragenyx
Case Series of Canadian Patients With Homozygous Familial Hypercholesterolemia Treated With ANGPTL3 Inhibitor Evinacumab (W-181) - May 11, 2023 - Abstract #ENDO2023ENDO_2607;
Five HoFH patients with distinctive histories, baseline treatments and ASCVD have all shown marked improvement in LDL-C levels with a mean reduction of 49.4% on evinacumab on top of existing therapy. Overall, observations from our case series suggest that evinacumab is an effective new treatment for HoFH, a life-threatening condition with few therapeutic options.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Evkeeza (evinacumab-dgnb) / Regeneron, Ultragenyx
Review, Journal: Current Treatment Options in Homozygous Familial Hypercholesterolemia. (Pubmed Central) - Jan 22, 2023
We are at the dawn of a new era of effective pharmacotherapies for HoFH patients, with new agents providing an LDL-receptor independent cholesterol reduction. In this context, the present review provides a summary of the currently available therapies and emerging therapeutic agents for the management of patients with HoFH, in light of recent evidence and guideline recommendations.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Nexletol (bempedoic acid) / Esperion Therap, Otsuka, Daiichi Sankyo
Review, Journal: A stepwise approach to prescribing novel lipid-lowering medications. (Pubmed Central) - Dec 17, 2022
Several new lipid lowering medications have received approval by regulatory agencies in the past decade including proprotein convertase subtilisin/kexin type 9 modulators, ATP-citrate lyase inhibitors, angiopoietin-like 3 inhibitors, lomitapide, and icosapent ethyl...This information can help the clinicians to prescribing these novel lipid lowering medications to achieve treatment goals and reduce the risk of atherosclerotic cardiovascular disease. The aim is to shift the paradigm for high-intensity statins from watch and wait to initial combination therapy for high-risk patients.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion
Enrollment change, Trial completion date, Trial termination, Trial primary completion date: Global Lomitapide Pregnancy Exposure Registry (clinicaltrials.gov) - Dec 16, 2022
P=N/A, N=5, Terminated,
The aim is to shift the paradigm for high-intensity statins from watch and wait to initial combination therapy for high-risk patients. N=30 --> 5 | Trial completion date: Mar 2028 --> Dec 2022 | Enrolling by invitation --> Terminated | Trial primary completion date: Mar 2028 --> Dec 2022; The PER objective has been integrated into the LOWER protocol.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion
Journal: Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. (Pubmed Central) - Dec 16, 2022
N=30 --> 5 | Trial completion date: Mar 2028 --> Dec 2022 | Enrolling by invitation --> Terminated | Trial primary completion date: Mar 2028 --> Dec 2022; The PER objective has been integrated into the LOWER protocol. These data indicate that the hepatic safety of lomitapide remains favourable with no clinically significant elevations in hepatic biomarkers and hepatic stiffness remained normal for up to 9.0 years follow-up.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Evkeeza (evinacumab) / Regeneron, Ultragenyx
Journal: New algorithms for treating homozygous familial hypercholesterolemia. (Pubmed Central) - Nov 10, 2022
Hypercholesterolemia is notoriously difficult to control in most patients with HoFH, but multi-LLT, including newer drugs, allows reduction of LDL-C to levels unimaginable until a few years ago. Cost and availability of these new therapies are important future challenges to be addressed.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Evkeeza (evinacumab) / Regeneron, Ultragenyx, Repatha (evolocumab) / Amgen, Astellas
Journal: Homozygous familial hypercholesterolaemia in a patient presenting with hypertensive encephalopathy. (Pubmed Central) - Nov 7, 2022
A significant proportion of patients with HoFH continue to have elevated LDL-C levels, thereby requiring second-line agents, such as proprotein convertase subtilisin/kexin type inhibitors (evolocumab), microsomal triglyceride transfer protein inhibitors (lomitapide) and angiopoietin-like protein inhibitors (evinacumab). This case report aimed to raise awareness among paediatricians to consider HoFH as a possible aetiology in a child presenting with hypertension and suggestive physical findings.
- |||||||||| Journal: Lipid-lowering drugs. (Pubmed Central) - Sep 20, 2022
Larger studies are warranted to determine lomitapide effectiveness in FCS. No abstract available
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion
Journal, PARP Biomarker, IO biomarker: Disruption of Lipid Raft Microdomains, Regulation of CD38, TP53, and MYC Signaling, and Induction of Apoptosis by Lomitapide in Multiple Myeloma Cells. (Pubmed Central) - Aug 28, 2022
Lomitapide is effective and safe in ARH therapy as well as in classical HoFH. Lomitapide treatment led to disruption of lipid raft domains and induction of pro-apoptotic factors and might, therefore, be considered as a potential therapeutic agent in MM.
- |||||||||| Review, Journal: Current Options and Future Perspectives in the Treatment of Dyslipidemia. (Pubmed Central) - Aug 27, 2022
This narrative review provides an overview of the new drugs for the treatment of dyslipidemia, their current status, ongoing clinical or preclinical trials, and their prospects. We also discuss the new alternative therapies for the treatment of dyslipidemia and their relevance to practice.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Evkeeza (evinacumab) / Regeneron, Ultragenyx, Nexletol (bempedoic acid) / Esperion Therap, Otsuka
Journal: Advancements in the Treatment of Homozygous Familial Hypercholesterolemia. (Pubmed Central) - Aug 4, 2022
For patients who cannot achieve adequate LDL-C reduction, lipoprotein apheresis may be necessary, with the added benefit of reducing lipoprotein(a) levels that carries an added risk if also elevated in patients with HoFH. Finally, gene therapy and genome editing using CRISPR/Cas-9 are moving through clinical development and may dramatically alter the future landscape of treatment for HoFH.
- |||||||||| Leqvio (inclisiran) / Alnylam, Novartis
Journal: Inclisiran: A Novel Small Interfering RNA Drug for LDL Reduction. (Pubmed Central) - Aug 3, 2022
Thus, inclisirin is a novel siRNA molecule that provides further options in the management of hypercholesterolemia refractory to statins alone. However, cost and evidence considerations suggest it should not supplant adjunctive therapy with the PCSK9 inhibiting monoclonal antibodies, despite having an efficacy, safety, tolerability, and drug interaction profile superior to other antihypercholesterolemic options such as lomitapide, niacin, bile acid sequestrants, and bempedoic acid.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Tasigna (nilotinib) / Novartis, Inhibikase
Journal: Integration of In Silico Strategies for Drug Repositioning towards P38α Mitogen-Activated Protein Kinase (MAPK) at the Allosteric Site. (Pubmed Central) - Jul 29, 2022
Key influential amino acids were mostly hydrophobic residues, while the two charged residues including E71 and D168 were considered crucial ones due to their ability to form very strong H-bonds with the focused drugs. Altogether, our contributions obtained here could be theoretical guidance for further conducting experimental-based preclinical studies necessary for developing therapeutic agents targeting p38α MAPK.
- |||||||||| Juxtapid (lomitapide) / Amryt, Novelion, Evkeeza (evinacumab) / Regeneron, Ultragenyx, Repatha (evolocumab) / Amgen, Astellas
Review, Journal: The Role of Non-statin Lipid-Lowering Medications in Youth with Hypercholesterolemia. (Pubmed Central) - May 3, 2022
A clinical trial for lomitapide is in progress. Approvals for PCSK9 and ANGPTL3 inhibitors have expanded opportunities for children and adolescents with HeFH and HoFH to achieve lower LDL-C levels.
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