Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028

Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028

Summary

SMA is an area that is expected to witness strong growth in the future, primarily due to launch of pipeline products that are expected to gradually capture market share from Biogen’s Spinraza. Also, the launch of survival motor neuron (SMN)- independent therapies are expected to be used in conjunction with an SMN-dependent therapy and this is also expected to contribute to SMA market growth in the forecast period.

Established pharmaceutical companies as well as smaller biotechnology companies are leading the development of pipeline products.

Developmental challenges include the need for long-term safety and clinical data for SMA drugs especially those that are proposed to work by gene-therapy, the need for early diagnosis of disease to ensure that the treatment has the maximum therapeutic effect, and the need to gain more understanding of the natural history of the disease by sharing data from previous trials and studies that may not have met the end-points but the results are capable of providing valuable insights on tried and tested methodologies in an effort to streamline various product development efforts.

Key Highlights

The SMA market is expected to witness a CAGR of 16.0% from 2018 to 2028, reaching global value of $6.6 billion.

GlobalData anticipates that four pipeline drugs will launch over the forecast period in the US, the EU, and Japan. These pipeline drugs are expected to address significant unmet needs for SMA and significantly boost growth in the SMA market across the 7MM.

Among these pipeline products, two products are similar in its MOA as Biogen’s Spinraza which was the first approved therapy for SMA. Novartis’ Zolgensma, a gene therapy which received FDA approval in May 2019, is also anticipated to receive regulatory approvals in the EU and Japan. This therapy is proposed to be administered as a one-off infusion and is one that has excited all the KOLs interviewed by GlobalData. The aforementioned drugs are proposed to address the root cause of the disease. In addition to these, there is another set of drugs in pipeline development that are aimed at improving muscle functions in SMA patients.

While clinical unmet needs such as need for more treatment options are expected to be met by launch of pipeline products in the forecast period, environmental unmet needs such as market access barriers are expected to be a prominent topic of discussion among pharmaceutical companies and funding agencies and one that will evolve over the forecast period to help develop sustainable funding mechanisms for expensive drugs.

Key Questions Answered

- What are the key unmet needs in SMA? What do KOLs identify as the key unmet needs? Will the products in the current pipeline fill these unmet needs?
- What were the key SMA treatments in 2018?
- When will the late stage pipeline products launch, and how will it affect drug sales and the overall SMA market in the 7MM?

Scope

- Overview of SMA, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
- Market Data including annualized SMA therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in fours patient segments (Type 1, 2, 3, and 4) forecast from 2018 to 2028.
- Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the SMA therapeutics market.
- Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for SMA therapy. The most promising candidates in Phase II and III development are profiled.
- Analysis of the current and future market competition in the global SMA therapeutics market: Insightful review of the key industry drivers and barriers. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

The report will enable you to:
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global SMA therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global SMA therapeutics market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.