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NVS SMA Drug Gets CHMP Backing, Malaria Drug Wins WHO Prequalification
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Key Takeaways
NVS gains CHMP support for Itvisma to treat SMA patients aged two years and older in the EU.
Itvisma showed significant motor function gains in the STEER study, meeting key endpoints.
NVS wins WHO prequalification for Coartem Baby, the first malaria drug for infants and newborns.
Novartis (NVS - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion recommending marketing authorization for Itvisma (intrathecal onasemnogene abeparvovec) for the treatment of individuals aged two years and older, living with 5q spinal muscular atrophy (SMA) caused by survival motor neuron 1 (SMN1) gene mutations.
The European Commission’s (EC) final decision is expected within two months. If approved, Itvisma would be the first and only gene replacement therapy for the specified age group with SMA in the region.
Separately, Novartis announced that the WHO has prequalified Coartem Baby (artemether-lumefantrine), the first antimalarial specifically designed for newborns and infants weighing 2 to 5 kilograms (kg).
Year to date, NVS’ shares have rallied 5.5% against the industry’s 6.5% decline.
Image Source: Zacks Investment Research
CHMP Nod for NVS’ Itvisma Backed by Multiple Study Data
CHMP’s positive opinion recommending marketing authorization for Itvisma is supported by data from the registrational STEER study, phase IIIb STRENGTH and phase I/II STRONG studies.
In the STEER study, Itvisma demonstrated a statistically significant 2.39-point improvement in motor function, with effects sustained more than 52 weeks of follow-up, compared with a 0.51-point improvement in the Sham group. The study met its primary endpoint.
The STEER and STRENGTH studies also showed clinically meaningful benefits for treatment-naïve and previously treated patients.
More on Novartis’ Itvisma
Itvisma is a one-time fixed-dose gene therapy designed to address the root cause of SMA by replacing the SMN1 gene, potentially improving motor function and reducing reliance on ongoing treatments.
The brand name Itvisma has been conditionally accepted by the EMA for the investigational therapy OAV101B (onasemnogene abeparvovec). While it has not yet received full approval from the EC, the FDA approved the therapy in November 2025 under the same brand name for the treatment of patients aged two years and older with SMA.
Spinal Muscular Atrophy is a rare inherited condition caused by a missing or defective SMN1 gene, which normally produces the SMN protein essential for muscle function, including breathing and movement. Without sufficient SMN protein, motor neurons progressively die, leading to worsening muscle weakness.
Novartis holds exclusive global licensing agreements with Nationwide Children's Hospital, REGENXBIO and Généthon covering AAV-based gene replacement technologies and delivery methods for treating all types of SMA.
WHO Prequalifies NVS’ Coartem Baby for Malaria
Concurrently, Novartis announced that its Coartem Baby has been prequalified by the WHO.
NVS developed Coartem Baby in collaboration with Medicines for Malaria Venture. The therapy is also called Riamet Baby in some regions.
Novartis plans to supply it largely on a not-for-profit basis in malaria-endemic countries.
The WHO pre-qualification program evaluates the quality, safety and efficacy of treatments for diseases such as malaria, HIV/AIDS and tuberculosis. Its output, including lists of pre-qualified products, serves as a trusted reference for the United Nations and other procurement agencies, helping guide funding and purchasing decisions in the public sector.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.87. Over the same period, EPS estimates for 2027 have surged from $2.85 to $3.25. CPRX shares have gained 17.3% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.94 to $3.00. Over the same period, EPS estimates for 2027 have surged from $3.27 to $3.29. INDV shares have lost 7.4% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ 2026 earnings per share have increased from $8.22 to $9.02. Over the same period, EPS estimates for 2027 have risen from $9.90 to $10.23. Year to date, shares of ANIP have lost 0.4%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
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NVS SMA Drug Gets CHMP Backing, Malaria Drug Wins WHO Prequalification
Key Takeaways
Novartis (NVS - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion recommending marketing authorization for Itvisma (intrathecal onasemnogene abeparvovec) for the treatment of individuals aged two years and older, living with 5q spinal muscular atrophy (SMA) caused by survival motor neuron 1 (SMN1) gene mutations.
The European Commission’s (EC) final decision is expected within two months. If approved, Itvisma would be the first and only gene replacement therapy for the specified age group with SMA in the region.
Separately, Novartis announced that the WHO has prequalified Coartem Baby (artemether-lumefantrine), the first antimalarial specifically designed for newborns and infants weighing 2 to 5 kilograms (kg).
Year to date, NVS’ shares have rallied 5.5% against the industry’s 6.5% decline.
Image Source: Zacks Investment Research
CHMP Nod for NVS’ Itvisma Backed by Multiple Study Data
CHMP’s positive opinion recommending marketing authorization for Itvisma is supported by data from the registrational STEER study, phase IIIb STRENGTH and phase I/II STRONG studies.
In the STEER study, Itvisma demonstrated a statistically significant 2.39-point improvement in motor function, with effects sustained more than 52 weeks of follow-up, compared with a 0.51-point improvement in the Sham group. The study met its primary endpoint.
The STEER and STRENGTH studies also showed clinically meaningful benefits for treatment-naïve and previously treated patients.
More on Novartis’ Itvisma
Itvisma is a one-time fixed-dose gene therapy designed to address the root cause of SMA by replacing the SMN1 gene, potentially improving motor function and reducing reliance on ongoing treatments.
The brand name Itvisma has been conditionally accepted by the EMA for the investigational therapy OAV101B (onasemnogene abeparvovec). While it has not yet received full approval from the EC, the FDA approved the therapy in November 2025 under the same brand name for the treatment of patients aged two years and older with SMA.
Spinal Muscular Atrophy is a rare inherited condition caused by a missing or defective SMN1 gene, which normally produces the SMN protein essential for muscle function, including breathing and movement. Without sufficient SMN protein, motor neurons progressively die, leading to worsening muscle weakness.
Novartis holds exclusive global licensing agreements with Nationwide Children's Hospital, REGENXBIO and Généthon covering AAV-based gene replacement technologies and delivery methods for treating all types of SMA.
WHO Prequalifies NVS’ Coartem Baby for Malaria
Concurrently, Novartis announced that its Coartem Baby has been prequalified by the WHO.
NVS developed Coartem Baby in collaboration with Medicines for Malaria Venture. The therapy is also called Riamet Baby in some regions.
Novartis plans to supply it largely on a not-for-profit basis in malaria-endemic countries.
The WHO pre-qualification program evaluates the quality, safety and efficacy of treatments for diseases such as malaria, HIV/AIDS and tuberculosis. Its output, including lists of pre-qualified products, serves as a trusted reference for the United Nations and other procurement agencies, helping guide funding and purchasing decisions in the public sector.
Novartis’ Zacks Rank & Stocks to Consider
NVS currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals (CPRX - Free Report) , currently sporting a Zacks Rank #1 (Strong Buy), and Indivior Pharmaceuticals (INDV - Free Report) and ANI Pharmaceuticals (ANIP - Free Report) , which carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.87. Over the same period, EPS estimates for 2027 have surged from $2.85 to $3.25. CPRX shares have gained 17.3% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.94 to $3.00. Over the same period, EPS estimates for 2027 have surged from $3.27 to $3.29. INDV shares have lost 7.4% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ 2026 earnings per share have increased from $8.22 to $9.02. Over the same period, EPS estimates for 2027 have risen from $9.90 to $10.23. Year to date, shares of ANIP have lost 0.4%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.