FDA Commissioner Refers To Rare Disease Drug With Associated Morbidity: Analysts Are Divided If Makary Meant Regenxbio Or Uniqure’s Experimental Therapy

• In an interview, Makary defended the FDA's recent, stricter stance on rare disease drug approvals.
• He specifically mentioned a "product" where researchers drilled burr holes in patients' skulls to inject a therapy, which showed no benefit in a randomized trial and had associated morbidity.
• While Leerink believes Makary was referring to Regenxbio's RGX-121, Stifel thinks he was referring to uniQure AMT-130.

Shares of uniQure fell 32% on Thursday on the heels of comments by FDA Commissioner Marty Makary on CNBC on rare disease drug applications.

In an interview with CNBC, Makary defended the FDA's recent, stricter stance on rare disease drug approvals. He specifically mentioned a "product" where researchers drilled burr holes in patients' skulls to inject a therapy, which showed no benefit in a randomized trial and had associated morbidity.

“I have a lot of sympathy for those patients. There's nothing out there to offer them, but we're not going to go ahead and approve something like that that has morbidity associated with it,” he said.

uniQure’s AMT-130

Makary’s comments were deemed a reference to uniQure’s Huntington’s disease gene therapy AMT-130, sending shares spiralling.

In November, uniQure said that it no longer believes that the FDA considers data from an early-to mid-stage trial of its investigational gene therapy sufficient for approval.

The company recently met with the agency, and although the minutes of the meeting have not been received, the company believes that the FDA no longer considers the data from the AMT-130 trial adequate to provide primary evidence in support of an application for approval, it then said.

As a result, it is now unclear when the company would apply for approval of the gene therapy, it said. uniQure, however, added that it intends to communicate with the FDA to determine a path forward toward approval of the therapy.

Analysts Chip In

Leerink, however, noted that the comments seem to point to a drug that had been officially reviewed and rejected, and highlights that Makary also mentioned the drug had morbidity associated with it and there was no cure. The firm believes this is referring to Regenxbio's (RGNX) RGX-121, and not AMT-130.

The FDA refused to approve Regenxbio’s Hunter syndrome gene therapy earlier this month citing insufficient evidence of effectiveness. The company said it is planning to meet with the FDA to address the complete response letter, gather expert input and longer-term clinical data to better define the eligible patient population, and work toward resubmitting its application as quickly as possible.

Stifel Believes Differently

Stifel, meanwhile, said that it can see why uniQure's shares are lower on comments from FDA commissioner Makary. Makary was defensive of FDA's approach broadly, as he appeared to maybe refer to the AMT-130 program as an example of something his FDA didn't get behind.

To be clear, his specific commentary might/might not have been referring to AMT-130 since uniQure never received an actual complete response letter, the firm noted. While some seem to think Makary was referring to Regenxbio's (RGNX) RGX-121, this doesn't really make sense to Stifel since he describes a drug that didn't beat a control arm, and the Regenxbio study in Hunter was single-arm, it said.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around QURE stock  rose from ‘bearish’ to ‘bullish’ territory over the past 24 hours while message volume rose from ‘normal’ to ‘high’ levels.

Sentiment around RGNX stayed within the ‘neutral’ territory over the past 24 hours, while message volume stayed at ‘extremely low’ levels.

QURE stock has risen 45% over the past 12 months, while RGNX gained 25%. 

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