Table 1. Several major acquisitions and partnership deals in the orphan drug industry.

Buyer	Seller	Type of deal, year of transaction	Estimated value of the deal	Major rare disease assets acquired
Sanofi	Genzyme	acquisition, 2011	$20.1 billion	Gaucher's disease treatment Cerezyme and the Fabry disease drug Fabrazyme
Shire	Baxalta	acquisition, 2016	$32 billion	several approved orphan drugs for hemophilia and Gaucher's disease, along with a rich pipeline in rare diseases
Shire	Dyax	acquisition, 2015	$5.9 billion	several late-stage assets in HAE + early-stage pipeline
Shire	NPS Pharma	acquisition, 2015	$5.2 billion	Gattex for shore bowel syndrome, Natpara for hypoparathyroidism
Alexion	Synageva BioPharma	acquisition, 2015	$8.4 billion	Kanuma for LAL deficiency + a pipeline of rare disease programs
Alexion	Enobia	acquisition, 2011	$1.1 billion	Strensiq for hypophosphatasia
BioMarin	Prosensa	acquisition, 2014	$840 million	Disapersen for DMD
BioMarin	Zacharin	acquisition, 2013	upfront $10 million + milestone up to $134 million	small molecules for lysosomal storage disorders (preclinical stage) including MPS III, Tay Sachs and Sandhoff; proprietary SensiPro® platform
BioMarin	ZyStor	acquisition, 2010	upfront $22 million + milestone up to $93 million	ERT for the treatment of lysosomal storage disorders such as Pompe's disease
Pfizer	FoldRx Pharmaceutical	acquisition, 2010	undisclosed amount	Tafamidis for familial amyloid polyneuropathy
Roche	Trophos	acquisition, 2015	$0.5 billion	SMA
Biogen	Ionis (formerly ISIS)	partnering, 2013	$100 million upfront + $220 million in milestone payments.	ALS, SMA
Biogen	AGTC	partnering, 2015	$124 million upfront + up to $1.1 billion in milestone payments	XLRS and XLRP

ALS, amyotrophic lateral sclerosis; DMD, Duchenne's muscular dystrophy; ERT, enzyme replacement therapies; HAE, hereditary angioedema; LAL, lysosomal acid lipase; MPS, Mucopolysaccharidoses; SMA, spinal muscular atrophy; XLRP, X-linked retinitis pigmentosa; XLRS, X-linked retinoschisis.