The spinal muscular atrophy treatment market size is estimated to be USD 3,986.5 million in 2022 and is expected to witness a CAGR of 18.81% during the forecast period 2023-2033. Surge in initiatives to improve the alternatives for treating rare diseases and rising demand for pharmaceuticals including Nusinersen, Onasemnogene Abeparvovecare are some of the factors contributing to the market growth. Additionally, growing public awareness of innovative therapies and increase in cases of spinal muscular atrophy (SMA) are other factors supporting the market growth. However, lack of qualified experts and high price of diagnosis and treatment is expected to hinder the growth.

Increase in cases of spinal muscular atrophy (SMA) is expected to propel the market growth during the forecast period. The National Organization for Rare Disorders (NORD) estimates that approximately 1 in 10,000 people worldwide suffer with spinal muscular atrophy (SMA). As a result, one of the key drivers driving market growth is the rising incidence of SMA and the approval of gene therapy as a treatment for SMA.

Growing public awareness of innovative therapies is predicted to fuel the market growth during the projected period. For instance, in order to bring together the SMA community, including researchers, patients, and their caregivers, clinicians, and others, Cure SMA arranged the 2022 Annual SMA Conference in Anaheim in May 2021. A wide range of activities, including educational seminars, keynote presentations for researchers, and social events, were carried out during this conference. These gatherings give manufacturers the chance to meet and interact with individuals with SMA and learn about their unmet medical requirements.

Segmentation

By Type

The market is categorized into type 1, type 2, type 3, and type 4. In the global market, the type 1 segment accounted for the largest revenue share in 2022 owing to the high incidence. It is one of the most prevalent forms of SMA, affecting about 60.0% of all newborns born with the condition. Market expansion is anticipated to be fueled by companies’ growing focus on creating novel medications for the treatment of SMA patients. In 2019, a number of treatments, notably Zolgensma (onasemnogene abeparvovec-xioi), have received FDA approval for the treatment of type-1 patients. Additionally, during the projected period, the type-1 segment is anticipated to exhibit profitable expansion. Strong product pipelines and rising research activity are attributed with this growth. Market expansion is supported by the introduction of a new gene therapy product for treating SMA symptoms. For instance, the only gene therapy medicine with FDA approval for regulating disease development is Zolgensma, created by Novartis AG.

By Treatment

The market is divided into drug and gene therapy. In 2022, the drug segment accounted for the highest revenue share due to extensive availability of products. Recently, the FDA has approved two medications, Spinraza (nusinersen) and Evrysdi, for the treatment of SMA (risdiplam). The segment is expanding owing to the low price of drug research and development and the ease of access compared to gene therapy treatments. The gene therapy category is predicted to rise at the fastest CAGR during the projected period due to the rising use of gene therapy in the treatment of spinal muscular atrophy. Market expansion is supported by the introduction of a new gene therapy product for treating SMA symptoms. For instance, the only gene therapy medicine with FDA approval for regulating disease development is Zolgensma, created by Novartis AG.

By Drug

The market is segregated into zolgensma (AVXS-101), evrysdi, spinraza, others. In the global market, the spinraza segment accounted for the largest revenue share in 2022 owing to existence of favourable reimbursement policies. Adult and paediatric patients can be treated with the prescription drug Spinraza. Experienced healthcare experts administer it through intrathecal injection. Some nations, including Italy, Norway, the Netherlands, Romania, Croatia, and Poland, offer complete worldwide reimbursement for the cost of spinraza. The evrysdi drug segment is anticipated to witness significant expansion over the course of the projected period. The survival rate of SMA patients 2 months of age and older is anticipated to increase with the use of this drug. Several governmental and commercial insurance providers, including Highmark (Medicare), Aetna, Anthem, BCBS Alabama, Cigna, Florida Blue (BCBS FL), HealthPartners, United Healthcare, Colorado, Connecticut, and HCSC, currently cover Evrysdi for the treatment of patients with SMA.

By Route of Administration

The market is segmented into injection and oral. In 2022, the injection segment accounted for the largest revenue share owing to the high target specificity and effectiveness related to this method of administration. The majority of SMA drugs on the market are administered intravenously by trained medical professionals. For instance, patients are given the prescribed dose of 12 mg (5mL) of SPINRAZA intrathecally. Additionally, a 5.5ml dose of Zolgensma is also given intravenously to the patient. The oral segment is anticipated to exhibit the fastest growth throughout the forecast period Due to its benefits over other route of administration methods, such as non-invasiveness, ease of digestion, pain avoidance, versatility, and low side effects. Only Evrysdi is currently FDA-approved for use in treating paediatric SMA patients with oral administration.

Regional Markets

In 2022, North America region accounted for the highest revenue in the spinal muscular atrophy treatment market and is expected to maintain its dominance during the forecast period. This is attributed to the rising prevalence of SMA disorders and growing spending on treatment supplies. For instance, approximately 10,000 and 25,000 children and adults live with SMA in the United States. Additionally, as of July 2022, there were 30 patients receiving Spinraza, and SMA Canada estimates that 700 persons in Canada had this syndrome overall. The market in Asia Pacific is anticipated to exhibit significant CAGR during the projection period. The introduction of new players in the area is accountable for the region’s expansion. For instance, the Japanese Ministry of Health, Labour and Welfare (MHLW) in March 2020 approved the Novartis AG gene therapy candidate Zolgensma (onasemnogene abeparvovec) for the treatment of paediatric patients in Japan. The market expansion in the region is anticipated to benefit from this approval.

Competitor Insights

Some of the key players operating in the spinal muscular atrophy treatment market are Cytokinetics, Novartis AG, Ionis Pharmaceuticals Inc., Biogen, Biohaven Pharmaceuticals, PTC Therapeutics, F. Hoffmann-La Roche Ltd, Scholar Rock, Inc., and NMD Pharma A/S.

To achieve a competitive edge, these companies are implementing a variety of growth strategies. Strategic partnerships, alliances, and agreements with different businesses and research institutions are seen to be the main strategies used by companies in this sector.

• For instance, Biogen and Ionis Pharmaceuticals, Inc., enter into a licensing agreement in January 2022 for the development of BIIB115 (an antisense oligonucleotide), an additional research medication, for the treatment of patients with SMA.

This comprehensive research report focuses on the global and regional market size and forecasts of diverse segments including type, treatment, drug, and route of administration from 2023 to 2033.

Report Scope:

Segmentation: Spinal Muscular Atrophy Treatment Market Report 2022 – 2033

Type (Revenue, USD Million), 2022 – 2033

• Type 1
• Type 2
• Type 3
• Type 4

Treatment (Revenue, USD Million), 2022 – 2033

• Drug
• Gene Therapy

Drug (Revenue, USD Million), 2022 – 2033

• Zolgensma (AVXS-101)
• Evrysdi
• Spinraza
• Others

Route of Administration (Revenue, USD Million), 2022 – 2033

• Injection
• Oral

By Region (Revenue, USD Million), 2022 – 2033

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • France
  • UK
  • Spain
  • Italy
  • Rest of Europe
• Asia Pacific
  • China
  • Japan
  • India
  • Australia
  • South Korea
  • Rest of APAC
• Latin America
  • Brazil
  • Mexico
  • Argentina
  • Rest of LATAM
• Middle East & Africa
  • South Africa
  • Saudi Arabia
  • Rest of MEA