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Daily Brief: GDPR and NHS Opt Out Day, BioMarin's new rare disease approval and more

It's not just the GDPR's big day, plus news from Medigene and Pfizer

Hello and welcome to another round up of news from pharma, biotech and healthcare.

GDPR and NHS opt-out day: do we have your consent?

Today is a red letter day in the world of data, as anyone with an email account will know, as most of us have been bombarded with messages from companies and organisations rushing to meet the new GDPR regulations.

These EU data privacy regulations gives consumers far greater clarity and control over how their personal data is handled by government organisations and businesses, and comes (coincidentally) in the wake of the Facebook and Cambridge Analytica data-scrapping scandal which has brought the issue to the fore globally.

Meanwhile, 25 May is also a very significant milestone for the NHS regarding data – today sees the introduction of the NHS ‘national data opt-out’. This allow individuals to opt-out from having their NHS medical records being used for research or planning purposes.

In this case ‘researchers’ could be working in universities, hospitals, medical royal colleges or be pharmaceutical companies researching new treatments.

This is NHS England’s second attempt at getting consent from the UK patients for wider use of their clinical data, after the bungled ‘’ consulation in 2014.  NHS England was heavily criticised for being seen to not properly explain that scheme, and was forced to abandon it and start again.

Today sees the re-launch of the idea- and NHS leaders will be hoping that as few as possible NHS registered patients choose to opt out.

By March 2020, patients will be able to use an online service to see how their data collected by NHS Digital has been used for purposes other than their direct care.

The data which the NHS holds is seen as one of its greatest advantages – other national health systems are fragmented by comparison, and the government believes the NHS could become a leader in digital health informatics if it can harness this.

Earlier this week, Theresa May unveiled a new vision for AI to allow far earlier diagnoses of cancers and other chronic conditions – a concept that also relies on access to anonymised records.


BioMarin sees blockbuster potential for newly-approved Palynziq

BioMarin has gained US approval for Palynziq (pegvaliase-pqpz) its second drug to treat adults patients with the rare condition phenylketonuria (PKU).

The company already has Kuvan on the market to treat the disease, but has gained a licence for Palynziq in patients whose condition is not being controlled on existing treatment.

Last year Kuvan earned the company $407 million, making it BioMarin’s second biggest selling product, but Palynziq promises to be even bigger.

Palynziq is a PEGylated recombinant phenylalanine ammonia lyase enzyme, and is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe, an amino acid which builds up to potentially toxic levels in these patients.

BioMarin has already announced that the drug will cost around $267,000 a year on average - this would put them well on course to achieve annual sales above $1bn. However the company could face resistance from US payers, who are increasingly seeking major discounts on high price treatments.

PKU is marked by an inability to break down Phe and left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, affecting how the individual thinks, feels, and acts.

Patients with PKU require life-long management, and must stick to a severely restrictive daily diet of medical foods and formula that avoids Phe, which is present in most foods.


Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin (pictured) welcome the approval, and said it was the culmination of more than a decade of work by BioMarin employees dedicated to helping patients with the condition.

Palynziq is expected to be available in the US by the end of June. Meanwhile, the company submitted the drug to the European Medicines Agency in March this year.


After bluebird expands deal, Medigene raises €32m

Having expanded its potentially lucrative deal with US biotech bluebird bio, T-cell specialists Medigene has raised an extra €32.3m ($37.8m) from private investors to help fuel its R&D pipeline.

Headquartered in Martinsried near Munich, Germany, the company is one of Europe’s most promising prospects in the fast-developing field of T-cell immunotherapy in cancer.

Just last week bluebird bio decided to expand its investments in Medigene’s T-cell receptor (TCR) immunotherapy platform, increasing the number of projects from four to six, and paying $8m upfront. Medigene could earn up to $500 million if both the two new targets reach the market, and the potential total value of the expanded deal now stands at more than $1.5 billion.

However there is still a long way to go, even to proof of concept, and the cancer immunotherapy field has become intensely crowded in the last few years.

Nevertheless, the expanded deal was a major vote of confidence in Medigene’s T-cell receptor (TCR) immunotherapy platform, and has allowed the firm to go back to investors for more cash.

Medigene raised the €32.3m through an oversubscribed private placement to institutional investors, with major shareholder QVT Financial LP also selling off some of its shares to help raise more funds for the biotech.

Dr. Thomas Taapken, Chief Financial Officer of Medigene, comments: “The strong demand in this offering clearly highlights investor interest in our maturing T cell cancer immunotherapy development pipeline. We are grateful for this strong demonstration of confidence in Medigene as a global leader in T cell immunology."

The German company is also working on two further novel platforms, Dendritic cell (DC) vaccines and T cell-specific monoclonal antibodies (TABS), with its most advanced candidate being a DC vaccine, WT-1/PRAME for acute myeloid leukaemia now in phase I.

A NICE ‘yes’ for Mylotarg – but two thirds of patients excluded

NICE has published draft guidance recommending Pfizer’s Mylotarg (gemtuzumab ozogamicin) in combination with chemotherapy for newly diagnosed de novo CD33-positive acute myeloid leukaemia (AML) – but only if the disease has favourable cytogenetics or where the cytogenetics are unknown because cytogenetic analysis was unsuccessful.

That means Mylotarg isn’t recommended for people whose disease has intermediate cytogenetic status, and Pfizer says this will exclude around 65% of the eligible patient population  the provisional recommendation.

Craig Eagle, Head of Oncology, Pfizer UK said: “this provisional recommendation currently excludes 65% of the eligible patient population and so we will continue working with NICE to help ensure that all patients who are eligible to benefit from this treatment are able to access it once a final recommendation is issued.”

NICE looks set to recommend another AML treatment, Novartis’ Rydapt (midostaurin), which is for newly diagnosed acute FLT3-mutation-positive myeloid leukaemia.

Article by
Andrew McConaghie

25th May 2018

From: Regulatory



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