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Daily Brief: Gottlieb sets out cell and gene pathway, NICE says no to Chiesi ultra-rare treatment and more

News from the frontline of pharma, biotech and healthcare

Welcome to another rapid round up of news from pharma, biotech and healthcare from Pharmaceutical Market Europe (PME).

A new era in haemophilia approaches

Yesterday’s big news came from two rival developing gene therapies which could represent a cure for haemophilia – Spark Therapeutics and BioMarin.

Spark provided more data to back up its SPK-9001 for patients with haemophilia B, showing the drug virtually eliminated bleeds in a group of 15 patients.

BioMarin meanwhile also provided long-term data on its haemophilia A treatment valrox.  While both gene therapies look to have good safety profiles, the big question is about the durability of the response, and doubts on this score saw BioMarin’s share price decline in response to its data.


Gottlieb’s vision for cell and gene therapy regulation


Meanwhile, FDA Commissioner Scott Gottlieb says cell and gene therapies will soon be the mainstay for treatment of many diseases, and has pledged to create a dedicated pathway for the cutting edge treatments.

This will be called the Regenerative Medicine Advanced Therapy designation (RMAT), and Gottlieb set out his vision for the pathway in a speech yesterday.

He says one key difference with advanced therapies is that the most of the regulatory scrutiny will be on product manufacturing and quality, rather than clinical efficacy, as in biologics or pharmaceuticals.

He also envisages a far greater need for efficacy to be proven post-marketing, and announced a new FDA investment in real world evidence.  Finally, the FDA is also looking at how it can help increase the yield from advanced therapy manufacturing, which is currently one of field’s greatest limiting factors.

Read the speech in full here


'Right to try' set to become law

Despite being opposed by most US patient groups, and pharma and biotech leaders, the controversial 'Right to Try' legislation was passed by the House of Representatives yesterday. President Trump is a strong advocate of the legislation, and is likely to sign it into law promptly. The law will give terminally ill patients access to drugs still in clinical trials and not approved by the FDA.

Despite voicing his own concerns about the plans in recent months, FDA Commissioner Scott Gottlieb says the regulator is now fully behind the plans and will play its part in implementing them.

Read more at STAT


NICE says no to Chiesi’s rare disease drug

England’s cost effectiveness watchdog NICE has published draft guidance not recommending Chiesi’s enzyme replacement therapy Lamzede (velmanase alfa).

The drug treats alpha-mannosidosis, a very rare and debilitating lysosomal storage disorder (LSD) that affects around 25 people in England.

The enzyme replacement therapy is given lifelong once a week by intravenous infusion at a dose of 1 mg/kg. The list price is £886.61 per 10 mg vial – producing a cost of hundreds of thousands of pounds a year for each patient.

The company agreed a commercially confident discount to the drug’s price with the Department of Health and Social Care, but NICE’s view is that

the clinical benefits are ‘highly uncertain both in the short and long term’ because of ‘important limitations/uncertainties in the trial evidence’ and says estimates of its cost effectiveness are much higher than the range NICE usually accepts for highly specialised technologies.


Celgene expands drug discovery alliance with Evotec

Celgene is experiencing a rollercoaster 2018, with late-stage drug setbacks, the departure of senior executives, and investor pressure to improve its once sector-leading growth prospects.

Last month the company's chief operating officer Scott Smith stepped down with immediate effect, with his responsibilities transferred to CEO Mark Alles. Smith looked to have paid the price for some costly slip-ups in recent months, most notably the FDA’s refused to review Celgene’s multiple sclerosis drug, ozanimod.

The company’s psoriasis drug Otezla is also underperforming, and another blockbuster candidate mongersen failed in phase III.

Celgene is cash rich, but its many investments are being watched closely by investors. Today it has announced a long-term strategic drug discovery and development partnership in oncology.

Evotec has built a reputation for its strength in drug discovery, and is becoming a partner of choice for pharma companies who want to outsource this expertise without partnering or acquiring a biotech.

The new partnership builds on the companies existing alliance in neurodegenerative diseases, but is clearly a longer term investment for Celgene, and won’t address near-term pressures for good news.

Article by
Andrew McConaghie

23rd May 2018

From: Marketing



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