Seven months after Alexion bagged the rare-disease drug Kanuma in its $8.4 billion buyout of Synageva, the FDA has come through with a marketing approval. The agency has now approved 41 new therapies in 2015, matching the chart-topping record for all of last year.
Alexion is working to expand beyond its reliance on rare disease drug Soliris but has again run into a recalcitrant cost regulator in the U.K. over one of its new orphan drugs. Yes, it drug Strensiq is effective at treating the very rare and crippling pediatric-onset hypophosphatasia but at a cost of more than half a million dollars a year, it just can't be justified, the National Institute for Health and Care Excellence (NICE) told the Cheshire, CT-based drugmaker.
Alexion's $1.08 billion deal to buy Enobia has paid off with an FDA approval of the "breakthrough" therapy that it gained in the blockbuster bargain. The FDA announced Friday afternoon that asfotase alfa--to be sold as Strensiq--won marketing clearance as a new therapy for extremely rare cases of a metabolic disease known as hypophosphatasia.
The year's 10 highest-paid development executives pulled in $124.4 million in total compensation, a roughly 35% jump over 2013's top earners. And while each entrant benefited from meeting individual company goals, the whole group benefited from biopharma's macroeconomic moment in the sun, as the value of stock awards skyrocketed alongside the industry index.
Despite the best efforts of Alexion, the FDA has lingering concerns about manufacturing of the drugmaker's orphan drug Soliris at a plant in Rhode Island and presented it with a Form 483 with new observations following a follow-up inspection of the plant last week.
For an idea of what kind of year a drug developer had, take a look at what it paid its head of R&D. Click through our roundup for a look at who ranks where in the biopharma R&D world. Read more >>
Alexion brought in a team of experts last year to help the company get on top of the manufacturing problems that led to an FDA warning letter for its plant in Rhode Island. But meanwhile, it has had problems with particulate tied to a contractor and today issued its third recall since August of orphan drug Soliris.
The FDA has been harping at the industry to make sure it gets its manufacturing right and Moderna Therapeutics, which is developing a way to enable the in vivo production of therapeutic proteins, intends to make sure it does.
Ireland's liberal tax code continues to attract drugmakers, but some of the long-term players are cutting back on their extensive manufacturing there. Bristol-Myers Squibb, with plans to lay off about 160 workers at two facilities, can now be added to that list.
It's fairly routine for the U.K.'s cost-effectiveness watchdogs to ask companies for more data to support approval for use by the country's National Health Service. Clinical data, price modeling, the works. But asking for information on R&D costs? That's something different altogether.