Alexion has been recognized mainly as the marketer of Soliris since 2007, but in just eight weeks the rare disease specialist added two new drugs to its commercial portfolio. Management also laid out an impressive early- to mid-stage pipeline during its first-ever investor day, suggesting the company is well positioned for long-term growth.

Biotechnology companies ended 2014 with $6bn in new venture capital in their pockets, which was the biggest annual total since 2007, but that's after private startups and early-stage businesses raised $2bn in the fourth quarter alone – the biggest single-quarter total in at least 19 years.

Alexion Pharmaceuticals said it has completed its rolling biologics license application submission to the FDA for asfotase alfa as a treatment for hypophosphatasia (HPP), a chronic and progressive ultra-rare metabolic disease, which can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.

As NIH funding becomes increasingly scarce, the La Jolla, Calif., research institute will use an anonymously donated gift to emphasize translational projects and take internal risk. Will it deliver returns that sustain the organization?