Alnylam completes enrollment of HELIOS-A phase III study of vutrisiran for the treatment of hATTR amyloidosis with polyneuropathy.
Alnylam Pharmaceuticals, Inc. announced that it has achieved full enrollment in its HELIOS-A Phase III study of vutrisiran an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. The study was designed to enroll 160 patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy across 68 sites in 22 countries. Alnylam is on track to report topline results from HELIOS-A in early 2021.
European Commission approves Beovu to treat wet age-related macular degeneration. Novartis
Novartis has announced the European Commission (EC) has approved Beovu (brolucizumab) injection for the treatment of wet age-related macular degeneration (AMD). Beovu is the first EC-approved anti-VEGF treatment to demonstrate superior resolution of retinal fluid (IRF/SRF), a key marker of disease activity, versus aflibercept (secondary endpoints). Beovu also offers the ability to start eligible wet AMD patients on a three-month dosing interval immediately after the loading phase.
Biologic efficacy for complications associated with moderate-to-severe psoriasis
This is the final part of a three-part series on treatments for different patient groups within moderate-to-severe psoriasis. In part 2, we discussed treatments that may be suitable for young women who are of childbearing age. Here we delve into the efficacy of TNF-α treatments in patients who present with more than one feature of psoriasis disease (for example, psoriatic arthritis and nail psoriasis) and for those who have failed on prior biologic treatment.
European Commission approves Sunosi to improve wakefulness and reduce excessive daytime sleepiness in adults with narcolepsy, Jazz Pharma
Jazz Pharmaceuticals plc announced that the European Commission approved Sunosi (solriamfetol) to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adults with narcolepsy (with or without cataplexy) or obstructive sleep apnea (OSA) whose EDS has not been satisfactorily treated by primary OSA therapy, such as continuous positive airway pressure (CPAP).
European Commission approval for Isturisa for the treatment of people with endogenous Cushing’s syndrome.- Recordati
Recodati‘s inhibitor of cortisol synthesis, Isturisa (osilodrostat), has been approved by the European Commission (EC) for the treatment of people with endogenous Cushing’s syndrome, the company announced. The decision follows a European Medicines Agency (EMA) committee recommendation in November to approve Isturisa for that purpose.
Alnylam Pharmaceuticals initiates a rolling submission to the FDA for lumasiran for the treatment of primary hyperoxaluria type 1 .
Alnylam Pharmaceuticals, Inc. has initiated a rolling submission of its New Drug Application (NDA) to the FDA for lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1 (PH1). The rolling submission allows...
Health Canada approves Rinvoq for treatment of rheumatoid arthritis patients who have had an inadequate response or intolerance to methotrexate (MTX).- AbbVie
AbbVie announced that Health Canada has approved Rinvoq (upadacitinib) for the treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate (MTX).
FDA approves Lynparza for the maintenance treatment of germline BRCA-mutated metastatic pancreatic adenocarcinoma AstraZeneca + Merck Inc.
AstraZeneca and MSD Inc. have announced that Lynparza (olaparib) has been approved in the US for the maintenance treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) metastatic pancreatic adenocarcinoma (pancreatic cancer) whose disease has not progressed on at least 16 weeks of a 1st-line platinum-based chemotherapy regimen. Patients will be selected for therapy based on an FDA-approved companion diagnostic for Lynparza.
Results of phase III SUNRISE 1 study of Dayvigo in insomnia published in JAMA Network Open journal.- Eisai
Eisai announced that JAMA Network Open (www.jamanetwork.com) published results of SUNRISE 1 (Study 304), a pivotal Phase III head-to-head study that compared Dayvigo (lemborexant) to placebo and an active comparator in patients with insomnia disorder.
Moderate-to-severe psoriasis: the disease burden and unmet needs in different patient groups
Welcome to part one of this three-part series comparing treatments for moderate-to-severe psoriasis. In this first part, we will discuss the unmet need and burden of disease in distinct patient groups, the pathophysiology of the disease and the treatment options currently available.
Update on AP 101 trial as a potential treatment for epidermolysis bullosa.- Amryt
Amryt announced the results of an unblinded interim efficacy analysis on its pivotal Phase III EASE trial for AP 101 as a potential treatment for Epidermolysis Bullosa ("EB").The analysis was conducted by an Independent Data Monitoring Committee ("IDMC") The IDMC recommended that the trial should continue with an increase of 48 patients in the study to a total of 230 evaluable patients, in order to achieve 80% statistical power.
How close are we to a disease-modifying treatment for Alzheimer’s?
Alzheimer’s disease is a snowballing problem, one that is currently without a convincing solution. The progressive decline in cognitive function seen in patients with Alzheimer’s disease is associated strongly with plaques formed of amyloid beta in the brain, creating neurofibrillary tangles and leading to neuronal destruction. While some treatments improve cognition in the short-term, current therapeutic options are unable to modify or slow the disease process.
Improving non-alcoholic steatohepatitis care – is the finishing line in sight?
An intense race is going on right now in non-alcoholic steatohepatitis (NASH) research. A number of significant players within the pharmaceutical and biotechnology sectors are competing to develop the first major therapeutic advance in treatment for this neglected condition. But should we consider it more of a relay race? Could improved diagnosis, disease assessment and even combination therapy be the key to victory over this increasingly prevalent disease?
Left ventricular assist devices – A viable therapeutic option for heart failure?
Alongside a pharmacological strategy to aid myocardial recovery, LVADs may prove to have a significant role to play in the ever-growing problem of heart failure. Although LVADs are not new onto the scene, the advance of technology – smaller, more efficient devices with better reliability and potentially transcutaneous power transfer – means we may be on the cusp of seeing many more deployed to tackle heart failure.
Antibody-drug conjugates (ADCs)
Over the past couple of decades, there have been advances in a class of drugs which for a long time was purely theoretical: antibody-drug conjugates (ADCs). The concept underlying ADCs is elegant – what if it were possible to ‘target’ particular groups of cells with a cytotoxic agent, rather than exposing every cell of the body to it?
It was the winner of the 1908 Nobel prize for medicine, Paul Ehrlich, who is first credited with discussing the concept of a “magic bullet”, able to target specific cells without damaging healthy tissue. With the advent of immunotherapy, as well as antibody-drug conjugates, we are beginning to realise this vision.
Results of inclisiran(ALN-PCSsc) from ORION-1 Phase II trial positive for the treatment of hypercholesterolemia. - The Medicines Company + Alnylam Pharma.
The Medicines Company and Alnylam Pharmaceuticals, Inc.announced positive results from the analysis of Day 90 data for 497 patients, as well as analysis of preliminary Day 180 data for 189 patients, enrolled in the ORION-1 Phase II study of inclisiran.
Phase III PHOENIX 1 study of Stelara (J&J Janssen Cilag) shows consistent results for plaque Psoriasis
New efficacy and safety data from the Phase III PHOENIX 1 study, one of two pivotal registration trials, showed that maintenance treatment with Stelara (ustekinumab), from J&J Janssen Cilag, for up to five years of follow up resulted in consistent, significant clinical response in adults with moderate to severe plaque Psoriasis