Statins ‘should be given to children with heart risks before the age of 10’

Statins 'should be given

Statins should be given to thousands of children by the age of 10 under radical new NHS guidance.

GPs are being urged to identify those who have an inherited risk of high cholesterol, amid warnings that the vast majority of cases are going undetected.

Estimates suggest up to 260,000 people – including 50,000 children – are suffering from genetic deficts which affect the body’s ability to break down cholesterol.

New guidance from the National Institute for Health and Care Excellence (Nice) today says statins should be offered to such cases, to reduce their risk of heart or stroke in midlife.

Just 15 per cent of those with the condition are being treated for it, Nice said, including just 600 of 56,000 children with the genetic problem.

Family doctors are being asked to trawl records to idenitfy those with very high cholesterol levels.

Where levels of more than 9 mmol/l are found in those over 30, and those of 7.5 mmol/l are found in those under 30, high-dose statins should be offered, the NHS guidance states.

And it says gene tests should be used to find other family members – including those below the age of 10 – who are at such heightened risk that they should be put on medication.

Around 56,000 children are estimated to suffer from familial hypercholesterolaemia (FH), yet just 600 have been diagnosed, charities say.

The condition gives men a 50 per cent chance of suffering a heart attack or stroke before the age of 50, while women have a one in three chance by the age of 60.

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Orkambi Reduces Main Biomarker of CF, Vertex Says in Updated Results on Four Therapies

Orkambi Reduces

Orkambi (lumacaftor/ivacaftor) reduced levels of the main biomarker of the lung disease cystic fibrosis and improved the nutritional status of children with the condition, according to a Phase 3 clinical trial.

The results were part of a recent update that Vertex Pharmaceuticals provided on Orkambi and three of its other CF therapies, Kalydeco (ivacaftor), tezacaftor (VX-661) and VX-371.

Vertex conducted the Phase 3 trial (NCT02797132) of Orkambi to evaluate its effectiveness and safety in preschoolers with two copies of the CFTR gene’s F508del mutation. The 60 children were aged 2 to 5. Mutations of the gene cause CF by producing faulty versions of the CFTR protein.

An indication of Orkambi’s effectiveness in the trial was that it reduced the production of the children’s sweat chloride and improved their nutritional status.

A sweat test is the gold standard for diagnosing CF because people with the disease have more chloride in their sweat than those who don’t. As for nutrition, the thick mucus that CF produces in the digestive system can prevent patients from absorbing nutrients and fat properly, leading to difficulty gaining weight and slower growth. CF also produces the mucus in lungs and other organs.

The Phase 3 trial also showed that Orkambi was safe and that the children tolerated it well. Researchers reported no adverse events besides those seen in studies of patients aged 6 to 11.

Based on the promising results of the trial, Vertex plans to submit a New Drug Application on Orkambi to the U.S. Food and Drug Administration during the first quarter of 2018. It will also ask the European Medicines Agency to extend the therapy’s availability to very young children.

Another Phase 3 trial (NCT02412111) that Vertex conducted evaluated a combination of tezacaftor and Kalydeco’s ability to reduce respiratory problems in patients more than 12 years old.

The study included 151 participants at 68 sites in the United States, Canada, Australia, and the European Union. The patients had one copy of the F508del mutation and one copy of another CFTR mutation.

Eight weeks of treatment with the combo led to a negligible improvement in a measure of patients’ lung function known as forced expiratory volume in one second, or FEV1. This is the amount of air that people can forcefully blow out of their lungs in one second.

The combo did lead to a reduction in sweat chloride that was larger than Kalydeco generated alone, however.

Given the results, Vertex has decided not to continue pursuing regulatory approval for the combo. One reason is that most patients older than 12 are eligible to receive Kalydeco by itself.

The FDA is expected to make a decision by February 2018 on a related New Drug Application that Vertex has filed. That application involves using the tezacaftor-Kalydeco combo to treat patients aged 12 or older who carry two copies of an F508del mutation or one copy of an F508del mutation plus another mutation. The FDA is giving the request priority review.

European regulators are expected to decide whether to approve the combo therapy in the second half of 2018.

Vertex has completed enrolling children 12 to 24 months for another Phase 3 trial (NCT03277196) of Kalydeco. It will evaluate the therapy’s safety in children less than 2 years old with a CFTR gating mutation and an R117H mutation.

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All about biosimilars –from development to registration

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Biosimilars can be described as near-copies of an original pharmaceutical product that another company may have manufactured. These products are versions of an original or innovative product, but are officially approved.

There is a misconception that they are similar to or are the same as generics, but this is not so in reality. Generics differ from a biosimilars in one major sense: While a biosimilar is only highly similar to the original product (called the reference product); a generic is the exact replica of the original, whose chemical structure it takes. Further, there is a difference in the ways they are manufactured, their complexity (generics are small and biosimilars, big), and the data each is required to furnish for gaining approval.

The regulatory requirements for gaining approval for biosimilars are considered complex. Different approaches are used in different regions around the world.

Exploration of all aspects of biosimilars

All about biosimilars –from development to registration

Professionals in the areas of biological sciences, who deal with biologics, need to have complete knowledge of the whole host of issues associated with biosimilars, ranging from their manufacturing process to regulatory guidelines. Given the complex nature of the science and the approval process; lack of in-depth knowledge can result in their application getting rejected by the regulatory authorities.

It is to help avoid these situations that GlobalCompliancePanel, a leading provider of professional trainings for all the areas of regulatory compliance, will be organizing a two-day seminar. The aim of this seminar is to offer complete understanding of the topic of biosimilars, from its clinical aspects to what it takes to gain approval, and a whole host of other issues relating to biosimilars.

The Director of this seminar is Salma Michor, Founder and CEO of Michor Consulting Schweiz GmbH. Salma teaches regulatory affairs and clinical strategies at the University of Krems, Austria, and is an independent expert to the European Commission. Michor Consulting Schweiz’s clients include Johnson & Johnson, Novartis, Shire, Pfizer and Colgate Palmolive. To gain the immense benefits this seminar offers, please register by visiting  All about biosimilars –from development to registration This course has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion.

Discussion of all the areas relating to biosimilars

The FDA’s approval process for biosimilars is quite complex. If a biosimilar product has to gain FDA approval, it has to show that it is highly similar to an FDA-approved reference product. It has to show that the biosimilar product has no substantial deviation from the reference product in terms of effectiveness and safety. If there are differences, the company has to show that these are within the permissible limits allowed for the product.

The Affordable Care Act of 2010 made a few amendments into the way the Public Health Service Act (PHS Act) creates an abbreviated licensure pathway for biosimilars which are “biosimilar” to or are “interchangeable” with an FDA-approved biological product. This has to be in the manner described in the Biologics Price Competition and Innovation Act (BPCI Act).

An interchangeable biosimilar product is one that can be interchangeable with the reference product. That is, it can be substituted for the reference product without the advice of the prescriber. This means that the interchangeable biosimilar has met the FDA-approved reference product’s standards, as well as additional standards required for it to show interchangeability.

Complete understanding of the process relating to biosimilars

All about biosimilars –from development to registration1

Over the two days of this seminar, Salma will help participants understand all the aspects relating to biosimilars. She will explain the challenges and choices that professionals in the field of biosimilars are up against. Biosimilar legislation in the US and the EU, the ways of establishing Target Product Profile (TPP) for a biosimilar, preclinical aspects, and quality, stability and preclinical testing will all be covered.

The Director will also lead participants into an exploration of the Phase I and Phase III clinical aspects relating to biosimilars, the existing guidelines on clinical testing and safety, registration process under the EMA, and market access for biosimilars in the US and Europe. She will intersperse these topics with relevant case studies.

This two-day seminar will offer value to those who deal with biosimilars in their work. These include Regulatory Affairs, Medical Officers and Clinical Trial Managers.

Salma will cover the following areas at this seminar: