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Zevra Stock Surges Close to 70% in 3 Months: Here's Why
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Zevra Therapeutics, Inc. (ZVRA - Free Report) obtained FDA approval for arimoclomol capsules for the treatment of Niemann-Pick disease type C (NPC) in September. The drug will be marketed under the brand name Miplyffa.
The FDA approved Miplyffa for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients aged two years and above.
Following the FDA nod, Miplyffa became the first treatment to be approved for NPC, an ultra-rare and progressive neurodegenerative disease.
Additionally, Zevra received a rare pediatric disease priority review voucher in conjunction with the FDA approval for Miplyffa.
Shares of Zevra have surged 68.5% in the past three months compared with the industry’s rise of 9.3%.
Image Source: Zacks Investment Research
The FDA approval for Miplyffa can be attributed as the reason for the stock price rally as the drug became the first approved treatment for this rare and devastating disease.
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.
ZVRA's Miplyffa Approval Comes After Certain Setbacks
The approval of Miplyffa comes after a series of setbacks faced by Zevra previously.
Please note that the FDA extended the review period for the new drug application (NDA) for arimoclomol in March 2024. The NDA, resubmitted in December 2023, was originally assigned a target action date of June 21, 2024.
The FDA 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.
In August 2024, the FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of the data supporting arimoclomol as an effective treatment for NPC.
The NDA for arimoclomol was then assigned a target action date of Sept. 21, 2024.
Miplyffa is likely to be launched in the United States in eight to 12 weeks following the FDA nod. The approval for Miplyffa should present a significant commercial opportunity for the company.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.53 to $4.81. Earnings per share estimates for 2025 have improved from $5.38 to $5.86. Year to date, shares of ANIP have increased 3%.
ANIP’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $1.91 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 41.8%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, with the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from 46 cents to 28 cents. Loss per share estimates for 2025 have narrowed from $1.67 to $1.14. Year to date, shares of FULC have plunged 45.7%.
FULC’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 393.18%.
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Zevra Stock Surges Close to 70% in 3 Months: Here's Why
Zevra Therapeutics, Inc. (ZVRA - Free Report) obtained FDA approval for arimoclomol capsules for the treatment of Niemann-Pick disease type C (NPC) in September. The drug will be marketed under the brand name Miplyffa.
The FDA approved Miplyffa for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients aged two years and above.
Following the FDA nod, Miplyffa became the first treatment to be approved for NPC, an ultra-rare and progressive neurodegenerative disease.
Additionally, Zevra received a rare pediatric disease priority review voucher in conjunction with the FDA approval for Miplyffa.
Shares of Zevra have surged 68.5% in the past three months compared with the industry’s rise of 9.3%.
Image Source: Zacks Investment Research
The FDA approval for Miplyffa can be attributed as the reason for the stock price rally as the drug became the first approved treatment for this rare and devastating disease.
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.
ZVRA's Miplyffa Approval Comes After Certain Setbacks
The approval of Miplyffa comes after a series of setbacks faced by Zevra previously.
Please note that the FDA extended the review period for the new drug application (NDA) for arimoclomol in March 2024. The NDA, resubmitted in December 2023, was originally assigned a target action date of June 21, 2024.
The FDA 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.
In August 2024, the FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of the data supporting arimoclomol as an effective treatment for NPC.
The NDA for arimoclomol was then assigned a target action date of Sept. 21, 2024.
Miplyffa is likely to be launched in the United States in eight to 12 weeks following the FDA nod. The approval for Miplyffa should present a significant commercial opportunity for the company.
Zevra Therapeutics, Inc. Price
Zevra Therapeutics, Inc. price | Zevra Therapeutics, Inc. Quote
ZVRA's Zacks Rank & Stocks to Consider
Zevra currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are ANI Pharmaceuticals, Inc. (ANIP - Free Report) , Krystal Biotech, Inc. (KRYS - Free Report) and Fulcrum Therapeutics, Inc. (FULC - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.53 to $4.81. Earnings per share estimates for 2025 have improved from $5.38 to $5.86. Year to date, shares of ANIP have increased 3%.
ANIP’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $1.91 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 41.8%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, with the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from 46 cents to 28 cents. Loss per share estimates for 2025 have narrowed from $1.67 to $1.14. Year to date, shares of FULC have plunged 45.7%.
FULC’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 393.18%.