Our pipeline
We focus our development resources on projects within our core therapeutic areas of Neuroscience, Rare Diseases, Gastrointestinal and Regenerative Medicine, as well as early development projects in additional therapeutic areas.
Total R&D expenditures of $965.5 million, $770.7 million and $661.5 million were incurred in the years ended December 31, 2012, 2011 and 2010 respectively.
| Disease Area | Project | Development Status | Territorial rights |
| Neuroscience | |||
| ADHD | INTUNIV (extended release guanfacine) | Registration in Canada (Regulatory submission in Q4 2011) | Global |
| ADHD in children and adolescents | INTUNIV | Phase 3 in EU (entered Phase 3 in Q2 2010) | Global |
| Adjunctive therapy in Major Depressive Disorder (MDD) | LDX (lisdexamfetamine dimesylate) | Phase 3 in US (entered Phase 3 in Q4 2011) | Global |
| Binge Eating Disorder (BED) in adults | LDX (lisdexamfetamine dimesylate) | Phase 3 in US (entered Phase 3 in Q4 2012) | Global |
| ADHD in children and adolescents | LDX (lisdexamfetamine dimesylate) | Phase 2-ready in Japan | Global(1) |
| ADHD in children and adolescents | INTUNIV | Phase 3-ready in Japan | Global(1) |
| Rare Diseases | |||
| ACE inhibitor-induced Angioedema | FIRAZYR | Registration in Europe Phase 3 ready in US | Global |
| Hunter Syndrome with CNS symptoms | HGT2310 | Phase 1/ 2 completed and preparation is underway for a Phase 3 trial | Global(2) |
| Sanfilippo A Syndrome (MPS IIIA) | HGT1410 | Phase 1/ 2 completed and preparation is underway for a Phase 2b trial | Global |
| Metachromatic Leukodystrophy (“MLD”) | HGT1110 | Phase 1/ 2 | Global |
| Gastrointestinal | |||
| Chronic Constipation (Males) | RESOLOR (prucalopride) | Phase 3 in EU (entered Phase 3 in Q4 2010) | Europe |
| Chronic Constipation | SPD555 (prucalopride) | Phase 3-ready in US | US |
| Refractory gastroesophageal reflux disease (“rGERD”) | SPD557 | Phase 2 | Europe and North America |
| Regenerative Medicine | |||
| Epidermolyis Bullosa (EB) | ABH001 | Phase 3 in US (entered Phase 3 in Q4 2012) | Global |
| Acute Vascular Repair | SRM003 | Phase 2 | Global |
| Other therapeutic area | |||
| Essential thrombocythaemia | XAGRID | Phase 3 in Japan (entered Phase 3 in Q4 2010) | Global |
| Dry eye disease | SPD606 (lifitegrast) | Phase 3 | Global |
| Chronic Iron-overload | SPD602 (FBS 0701) | Phase 2 | Global |
| Prevention of retinopathy of prematurity (ROP) | HGT-ROP-001 | Phase 2 | Global |
(1) Under co-development with Shionogi as a result of a license and collaboration agreement.
(2) Genzyme has rights to manage marketing and distribution in Japan and certain other Asia Pacific countries under a license with Shire.
