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Income Taxes - Components of the Company's Deferred Tax Assets (Liabilities) (Detail) (USD $) In Thousands, unless otherwise specified	Dec. 31, 2014	Dec. 31, 2013
Deferred income tax assets:
Federal U.S. net operating loss carryforward	$ 55,358us-gaap_DeferredTaxAssetsOperatingLossCarryforwardsDomestic	$ 36,561us-gaap_DeferredTaxAssetsOperatingLossCarryforwardsDomestic
State net operating loss carryforward	3,723us-gaap_DeferredTaxAssetsOperatingLossCarryforwardsStateAndLocal	3,316us-gaap_DeferredTaxAssetsOperatingLossCarryforwardsStateAndLocal
Research and development credit, net	7,177us-gaap_DeferredTaxAssetsTaxCreditCarryforwardsResearch	3,786us-gaap_DeferredTaxAssetsTaxCreditCarryforwardsResearch
Orphan drug credit, net	19,287us-gaap_DeferredTaxAssetsTaxCreditCarryforwardsOther	19,883us-gaap_DeferredTaxAssetsTaxCreditCarryforwardsOther
Deferred rent	4,069us-gaap_DeferredTaxAssetsTaxDeferredExpenseReservesAndAccrualsDeferredRent	5,132us-gaap_DeferredTaxAssetsTaxDeferredExpenseReservesAndAccrualsDeferredRent
Deferred revenue	3,989us-gaap_DeferredTaxAssetsDeferredIncome	8,217us-gaap_DeferredTaxAssetsDeferredIncome
Depreciation	1,337us-gaap_DeferredTaxAssetsPropertyPlantAndEquipment	1,438us-gaap_DeferredTaxAssetsPropertyPlantAndEquipment
Other	962us-gaap_DeferredTaxAssetsOther	1,439us-gaap_DeferredTaxAssetsOther
Gross deferred income tax assets	95,902us-gaap_DeferredTaxAssetsGross	79,772us-gaap_DeferredTaxAssetsGross
Valuation allowance	(94,297)us-gaap_DeferredTaxAssetsValuationAllowance	(79,377)us-gaap_DeferredTaxAssetsValuationAllowance
Net deferred income tax assets	1,605us-gaap_DeferredTaxAssetsNet	395us-gaap_DeferredTaxAssetsNet
Deferred income tax liabilities:
Prepaid expenditures	(1,605)us-gaap_DeferredTaxLiabilitiesPrepaidExpenses	(395)us-gaap_DeferredTaxLiabilitiesPrepaidExpenses
Gross deferred income tax liabilities	(1,605)us-gaap_DeferredIncomeTaxLiabilities	(395)us-gaap_DeferredIncomeTaxLiabilities
Net deferred income tax asset/(liability)	$ 0us-gaap_DeferredTaxAssetsLiabilitiesNet	$ 0us-gaap_DeferredTaxAssetsLiabilitiesNet

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