FDA, RNAi

FDA adds ‘breakthrough’ status for Alnylam as patisiran starts its victory lap at the agency

With Alnylam’s rolling application for its landmark RNA therapy patisiran just weeks from completion, the FDA has come through with a breakthrough drug designation that may well trim its time to market.

The Cambridge/Boston biotech reported this morning that the agency had issued its BTD — providing VIP status at the agency — based on some stellar Phase III data in hereditary transthyretin-mediated ATTR amyloidosis — or hATTR amyloidosis.

That will further help Alnylam position this drug for a launch, provided no nasty surprises await during the FDA review period. Alnylam $ALNY has been squaring off against rival Ionis $IONS, which has also racked up positive data for its hATTR amyloidosis drug.

Eric Green

Alnylam has been working for 15 years to get to this stage, standing on the threshold of a market launch that will transform the company. Its success marks a key milestone for the RNA field, offering important validation for the science after a long period that saw Big Pharma coming in, dropping out and then coming back in. In this case Sanofi will likely be feeling the glow. The giant signed off on a partnership with Alnylam that gives them ex-North American marketing rights.

Some analysts scoffed at the BTD when the FDA first adopted the title program for speeding reviews.  But it’s helped revolutionize the oncology field — speeding approvals — and appears to be gaining even wider traction in the wake of Scott Gottlieb’s confirmation as commissioner, vowing to speed up the process in all sectors.

“The robust evidence provided by the APOLLO Phase 3 study demonstrated the potential of investigational patisiran to transform the lives of people with hATTR amyloidosis,” said Eric Green, vice president and general manager of the TTR program. “Breakthrough Therapy Designation enables us to work closely with the FDA on the review of the NDA to potentially bring patisiran to patients living with this devastating disease as quickly as possible.”


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