Zevra Therapeutics Resubmits Arimoclomol NDA To FDA

RTTNews | Pred 583 dňami
Zevra Therapeutics Resubmits Arimoclomol NDA To FDA

(RTTNews) - Zevra Therapeutics, Inc. (ZVRA) announced Wednesday it resubmitted its New Drug Application (NDA) for arimoclomol to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Arimoclomol is an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC).

The new data included in the resubmission comes from multiple non-clinical studies, natural history comparisons, real-world data generated from the ongoing early access programs in the U.S. and the European Union. It also included data from the four-year open-label extension of the Phase 2/3 clinical trial (NCT02612129). Results from this open-label trial suggest that arimoclomol reduces the long-term progression of NPC.

Arimoclomol has been evaluated in a total of 21 studies across a range of Phase 1, 2 or 3 clinical trials evaluating its safety and efficacy across more than 600 subjects in NPC, other disease or healthy subjects.

The primary efficacy trial evaluating arimoclomol for the treatment of NPC was a Phase 2/3 double-blind, placebo-controlled trial (CT-ORZY-NPC-002) of arimoclomol in 50 patients with NPC.

Based on standard NDA resubmission review timelines, an acknowledgment letter from the FDA that the resubmission is complete and setting the PDUFA date is expected within 30 days. Zevra expects the NDA to be classified as a Class II submission which would be subject to a review period by the FDA within six months from the date of submission.

read more
Zevra Therapeutics Says FDA Extends NDA Review Period For Arimoclomol

Zevra Therapeutics Says FDA Extends NDA Review Period For Arimoclomol

Zevra Therapeutics, Inc. (ZVRA) announced Monday the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for arimoclomol, an investigational orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC).
RTTNews | Pred 515 dňami
U.S. Construction Spending Unexpectedly Sees Further Downside In June

U.S. Construction Spending Unexpectedly Sees Further Downside In June

The Commerce Department released a report on Friday unexpectedly showing a continued decrease by U.S. construction spending in the month of June. The report said construction spending fell by 0.4 percent to an annual rate of $2.136 trillion in June after sliding by 0.5 percent to a revised rate of $2.144 trillion in May.
RTTNews | Pred 1 h 1 min
U.S. Consumer Sentiment Improves Marginally Less Than Previously Estimated In July

U.S. Consumer Sentiment Improves Marginally Less Than Previously Estimated In July

Revised data released by the University of Michigan on Friday showed consumer sentiment in the U.S. improved marginally less than previously estimated in the month of July. The University of Michigan said its consumer sentiment index for July was downwardly revised to 61.7 from a preliminary reading of 61.8. Economists had expected the index to be upwardly revised to 62.0.
RTTNews | Pred 1 h 5 min
U.S. Manufacturing Index Unexpectedly Dips To Nine-Month Low In July

U.S. Manufacturing Index Unexpectedly Dips To Nine-Month Low In July

Manufacturing activity in the U.S. unexpectedly contracted at a slightly faster rate in the month of July, according to a report released by the Institute for Supply Management on Friday. The ISM said its manufacturing PMI edged down to 48.0 in July after inching up to 49.0 in June with a reading below 50 indicating contraction.
RTTNews | Pred 1 h 7 min
Eurozone Inflation Steady At 2% ECB Target

Eurozone Inflation Steady At 2% ECB Target

Euro area consumer price inflation was unchanged at the European Central Bank's 2 percent target in July, easing the pressure for more easing. The harmonized index of consumer prices increased 2.0 percent year-on-year in July, the same rate of increase as seen in June. Inflation was forecast to ease marginally to 1.9 percent.
RTTNews | Pred 1 h 54 min
Bay Street Likely To Open On Weak Note

Bay Street Likely To Open On Weak Note

Canadian shares are set to open on a negative note on Friday as U.S. President Donald Trump's announcement of fresh tariffs on Canada and several other countries has hurt investor sentiment across global markets.
RTTNews | Pred 3 h 14 min