Rhythm | December 01, 2020
Stoutness is a significant issue around the world, in any case, for a small amount of patients, hereditary changes instead of propensities are driving the sickness. With its new FDA endorsement for Imcivree, Rhythm Pharmaceuticals is venturing out treating those cases.
For a subset of heftiness patients, hereditary transformations leave them with an "voracious appetite," Rhythm CEO David Meeker, M.D., said in a meeting. Beginning stage hereditary illnesses can leave patients incapable to control corpulence with diet and exercise, and, presently, Rhythm has scored the main FDA endorsement for a focused on medication pointed toward treating them.
Imcivree, once known as setmelanotide, scored a FDA gesture to treat patients who have varieties of the POMC, PCSK1 or LEPR qualities. The medication focuses on the MC4R pathway answerable for directing yearning, and the three qualities remembered for the underlying endorsement are only an initial step, Meeker said. The organization built up a test that takes a gander at around 100 hereditary transformations, and it's trying the medication in other hereditary changes with eyes on expected endorsements down the line.
With obesity, “you can see the forest,” Meeker said, because the problem is everywhere. The challenge is finding patients who could have an underlying genetic mutations causing excessive hunger, he said, noting that “testing becomes key.”
Most people, and most doctors, still think of obesity as a “calories in, calories out,” problem, said Michael Dedekian, medical director of pediatric specialty care at Barbara Bush Children’s Hospital in Maine. Very quickly, though, the medical community’s understanding of obesity is deepening, he said. And, as that’s happening, it’s becoming clear that genetic defects can “wildly disregulate” patients’ hunger.
With the approval and new knowledge about genetic defects, Dedekian believes the medical community is entering a “whole new era of obesity treatment." Over time, he has no doubt awareness of the genetic mutations driving some cases will increase. When abnormal hunger presents itself in young children, doctors should strongly consider a genetic test, Dedekian said.
Rhythm isn’t planning to deploy a sales force for its initial genetic mutations and will instead focus on “intensive efforts” around disease education and making its test more available, Meeker said. When doctors are confronted with a patient with abnormal hunger, they should be “thinking deep” about the cause, he added.
IQVIA™ | September 14, 2021
IQVIA™ , a leading global provider of advanced analytics, technology solutions and clinical research services to the life sciences industry, today announced a transformative collaboration with NRx Pharmaceuticals (NRx). NRx is a company dedicated to creating innovative, life-saving treatments and bringing hope to those facing life-altering conditions with no approved disease-modifying therapies or cures. IQVIA is excited to collaborate with NRx as its designated partner, to provide pharmacovigilance services and medical information in preparation for potential regulatory actions.
"As the world suffers yet another surge of COVID-19, thousands of patients require intensive care and too many succumb to this virus. New medicines with new mechanisms of action are urgently needed," said Robert Besthof, chief commercial officer and head of Operations of NRx. "As a small biopharma company preparing to launch a lifesaving medicine across multiple countries, we deeply appreciate the global reach that IQVIA is able to offer in support of patients, families, and those who care for them.”
This collaboration will allow NRx to access IQVIA’s domain experience with COVID-19, our unparalleled data assets, and analytics to support potential emergency use authorization (EUA) of ZYESAMI. IQVIA will work closely with NRx to support key activities required for EUA activation, including the pharmacovigilance and medical information programs.
“We are honored by the opportunity to join with NRx on the EUA activation and compliance for ZYESAMI in treating some of the sickest patients suffering from COVID-19,” said Susan Kitlas, vice president, Precommercial Business Unit, IQVIA. “IQVIA’s experience and expertise within the COVID-19 market will be a valuable component in helping make this treatment more widely available.”
NRx Pharmaceuticals draws upon more than 300 combined years of scientific and drug-development experience to bring improved health to patients. Its investigational product, ZYESAMI™ (aviptadil) for patients with COVID-19, has been granted Fast Track designation by the US Food and Drug Administration and is currently undergoing phase 3 trials funded by the US National Institutes of Health, the Biomedical Advanced Research and Development Authority part of the US Department of Health and Human Services, and the Medical Countermeasures program, part of the US Department of Defense.
IQVIA is a leading global provider of advanced analytics, technology solutions, and clinical research services to the life sciences industry. IQVIA creates intelligent connections across all aspects of healthcare through its analytics, transformative technology, big data resources and extensive domain expertise. IQVIA Connected Intelligence™ delivers powerful insights with speed and agility enabling customers to accelerate the clinical development and commercialization of innovative medical treatments that improve healthcare outcomes for patients. With approximately 74,000 employees, IQVIA conducts operations in more than 100 countries. IQVIA is a global leader in protecting individual patient privacy. The company uses a wide variety of privacy-enhancing technologies and safeguards to protect individual privacy while generating and analyzing information on a scale that helps healthcare stakeholders identify disease patterns and correlate with the precise treatment path and therapy needed for better outcomes. IQVIA’s insights and execution capabilities help biotech, medical device and pharmaceutical companies, medical researchers, government agencies, payers and other healthcare stakeholders tap into a deeper understanding of diseases, human behaviors and scientific advances, in an effort to advance their path toward cures.
Intravacc | September 08, 2020
Intravacc, a global leader in translational research and development of viral and bacterial vaccines, today announced that it has been awarded a contract with base and options that may total US$9.4 million from the US National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), for the development of a prophylactic vaccine against enterovirus D68 (EV D68). EV D68 is a respiratory virus that can cause childhood paralysis, Acute Flaccid Myelitis (AFM). Intravacc will develop an inactivated EV D68 vaccine, based on Intravacc's proprietary Vero cell technology, from early product selection through to Phase I clinical testing.