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Zevra (ZVRA) Rises on Update on Niemann-Pick Disease Type C Drug
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Zevra Therapeutics, Inc. (ZVRA - Free Report) announced that the FDA has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (“GeMDAC”) to review the company’s new drug application (NDA) for pipeline candidate arimoclomol.
The NDA is seeking approval for arimoclomol, an orally delivered, first-in-class treatment for Niemann-Pick disease type C (“NPC”).
NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissues and organs, including the brain.
Investors were pleased with this positive regulatory update and shares of the company rose 21.94% on Jul 9 on the news.
The FDA will hold the meeting with GeMDAC on Aug 2 to review the NDA.
The regulatory body intends to make the background materials available to the public no later than two business days before the meeting.
The NDA for arimoclomol has been assigned a target action date of Sep 21, 2024.
Please note that the FDA extended the review period for the NDA in March 2024. The NDA, resubmitted in December 2023, was originally assigned a target action date of Jun 21, 2024.
The FDA had issued a complete response letter in June 2021 in response to the prior arimoclomol NDA filing. Hence, the NDA was resubmitted, including additional evidence supporting trial metrics, FDA-preferred analyses, and data from multiple additional studies that provide supporting evidence of arimoclomol’s efficacy in clinical and non-clinical settings.
Zevra earlier reported that The National Niemann-Pick Disease Foundation, along with six other NPC advocacy and research organizations, submitted a petition in support of arimoclomol with nearly 1,000 signatures from NPC patients, caregivers and physicians.
A potential approval will expand Zevra’s portfolio and relieve investors.
Arimoclomol enjoys the Orphan Drug status in the United States. It has also received the Fast Track designation, Breakthrough Therapy designation and Rare Pediatric Disease designation from the FDA. The European Medicines Agency also granted the Orphan Medicinal Product designation to arimoclomol for the treatment of NPC.
Arimoclomol was added to Zevra’s pipeline following the acquisition of Orphazyme in 2022.
Shares of Zevra have lost 16.4% year to date compared with the industry’s decline of 7.9%.
Image Source: Zacks Investment Research
Zevra has a diverse portfolio of products and product candidates. It intends to leverage the commercial infrastructure that is in place for its other drug, Olpruva (sodium phenylbutyrate), to accelerate patient access for arimoclomol, set for a potential commercial launch in the United States upon approval.
Olpruva for oral suspension is approved by the FDA for the treatment of urea cycle disorders.
KP1077 is Zevra’s lead candidate, which is being developed as a treatment for idiopathic hypersomnia ("IH"), a rare neurological sleep disorder, and narcolepsy. The FDA has granted the orphan drug designation to KP1077 for the treatment of IH.
The company also has other candidates in the pipeline, including celiprolol, for the treatment of vascular Ehlers-Danlos syndrome in patients with a confirmed type III collagen mutation.
Entrada’s loss per share estimate for 2024 has narrowed from 27 cents to 13 cents in the past 60 days. The same for 2025 has narrowed from $3.44 to $3.21 in the past 30 days.
In the past 60 days, estimates for CORT’s 2024 earnings per share (EPS) have improved from 90 to 95 cents. Shares of CORT have rallied 24.5% in the past three months.
CORT’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 31.25%.
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Zevra (ZVRA) Rises on Update on Niemann-Pick Disease Type C Drug
Zevra Therapeutics, Inc. (ZVRA - Free Report) announced that the FDA has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (“GeMDAC”) to review the company’s new drug application (NDA) for pipeline candidate arimoclomol.
The NDA is seeking approval for arimoclomol, an orally delivered, first-in-class treatment for Niemann-Pick disease type C (“NPC”).
NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissues and organs, including the brain.
Investors were pleased with this positive regulatory update and shares of the company rose 21.94% on Jul 9 on the news.
The FDA will hold the meeting with GeMDAC on Aug 2 to review the NDA.
The regulatory body intends to make the background materials available to the public no later than two business days before the meeting.
The NDA for arimoclomol has been assigned a target action date of Sep 21, 2024.
Please note that the FDA extended the review period for the NDA in March 2024. The NDA, resubmitted in December 2023, was originally assigned a target action date of Jun 21, 2024.
The FDA had issued a complete response letter in June 2021 in response to the prior arimoclomol NDA filing. Hence, the NDA was resubmitted, including additional evidence supporting trial metrics, FDA-preferred analyses, and data from multiple additional studies that provide supporting evidence of arimoclomol’s efficacy in clinical and non-clinical settings.
Zevra earlier reported that The National Niemann-Pick Disease Foundation, along with six other NPC advocacy and research organizations, submitted a petition in support of arimoclomol with nearly 1,000 signatures from NPC patients, caregivers and physicians.
A potential approval will expand Zevra’s portfolio and relieve investors.
Arimoclomol enjoys the Orphan Drug status in the United States. It has also received the Fast Track designation, Breakthrough Therapy designation and Rare Pediatric Disease designation from the FDA. The European Medicines Agency also granted the Orphan Medicinal Product designation to arimoclomol for the treatment of NPC.
Arimoclomol was added to Zevra’s pipeline following the acquisition of Orphazyme in 2022.
Shares of Zevra have lost 16.4% year to date compared with the industry’s decline of 7.9%.
Image Source: Zacks Investment Research
Zevra has a diverse portfolio of products and product candidates. It intends to leverage the commercial infrastructure that is in place for its other drug, Olpruva (sodium phenylbutyrate), to accelerate patient access for arimoclomol, set for a potential commercial launch in the United States upon approval.
Olpruva for oral suspension is approved by the FDA for the treatment of urea cycle disorders.
KP1077 is Zevra’s lead candidate, which is being developed as a treatment for idiopathic hypersomnia ("IH"), a rare neurological sleep disorder, and narcolepsy. The FDA has granted the orphan drug designation to KP1077 for the treatment of IH.
The company also has other candidates in the pipeline, including celiprolol, for the treatment of vascular Ehlers-Danlos syndrome in patients with a confirmed type III collagen mutation.
Zacks Rank & Stocks to Consider
Zevra currently carries a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the healthcare sector are Entrada Therapeutics (TRDA - Free Report) and Corcept Therapeutics (CORT - Free Report) , both carrying a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Entrada’s loss per share estimate for 2024 has narrowed from 27 cents to 13 cents in the past 60 days. The same for 2025 has narrowed from $3.44 to $3.21 in the past 30 days.
In the past 60 days, estimates for CORT’s 2024 earnings per share (EPS) have improved from 90 to 95 cents. Shares of CORT have rallied 24.5% in the past three months.
CORT’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 31.25%.