FIRST: Roche launches Spinal Muscular Atrophy treatment Evrysdi in India

SMA is a severe, progressive rare neuromuscular disease that can be fatal. It affects approximately one in 10,000 live births globally and one in 7,744 live births in India and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

Evrysdi is administered daily at home orally (it is supplied as powder which is constituted into a liquid solution and taken once daily by mouth or feeding tube if required) and is designed to treat SMA by increasing production of the Survival Motor Neuron (SMN) protein. It is approved for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older.

Evrysdi is being studied in more than 450 people as part of the broadest, large and robust clinical trial program in SMA. The program included newborn infants to adults aged 60 years with varying symptoms and motor function, and is the only program that has included those that were previously treated for SMA with another medication.

Evrysdi was approved by Indian Health Authorities after reviewing its efficacy and safety data from three global clinical studies designed to represent a broad spectrum of people living with SMA:

1. FIREFISH3 in symptomatic infants aged 1 to 7 months with Type 1 SMA

2.SUNFISH3 in children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA.

Evrysdi showed clinically meaningful improvements in motor function across these two clinical trials in people with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA. Infants achieved the ability to sit without support for at least 5 seconds, a key motor milestone not normally seen in the natural course of the disease. Evrysdi also improved survival without permanent ventilation at 12 and 23 months, compared to natural history. Evrysdi maintained the ability to swallow and feed orally in the majority of the infants after 2 years of therapy. This is a unique characteristic of Evrysdi that was not observed with other medications for SMA.

3. JEWELFISH3 (interim data) is a study assessing people with any SMA type and disease severity who have previously been treated with other SMA targeted therapies between 6 months and 60 years. This study enrolled the broadest patient population ever studied in an SMA trial. Data from the JEWELFISH study, which included a diverse patient population with a high degree of motor impairment, show that Evrysdi has a favorable safety profile in patients previously treated with other SMA targeting therapies. Evrysdi also showed to improve SMN protein levels to 2 fold and above in all the patients who have received a previous therapy.

Evrysdi's clinical development program also includes:

RAINBOWFISH3 is a study assessing pre-symptomatic infants with Type 1 SMA from birth to 6 weeks old. Preliminary efficacy data from RAINBOWFISH showed that infants treated for 12 months achieved age appropriate motor milestones within the WHO windows for healthy children, including sitting, standing and walking, and improvements in motor function.

V Simpson Emmanuel, CEO and Managing Director, Roche Pharma India, says, "The launch of Evrysdi in India is a fine example of Roche living its purpose of 'Doing now what patients need next'. We are betting big on solving complex challenges related to rare diseases as we believe no patient should be deprived an opportunity to live a healthy life, however complex or rare the disease is. Today heralds a new journey of hope as we are all coming together to add color into the lives of SMA patients in India with the launch of Evrysdi, the first and only approved treatment in India for patients living with SMA. This also marks our foray into rare disease treatment in India."

To ensure every patient gets access to this disease modifying therapy, Roche is announcing its Patient Support program (PSP) for Evrysdi. Through this program:

• In the first two years of treatment, Roche provides three bottles free for every two bottles bought by the patient
• From the third year onwards, Roche provides two bottles free for every one bottle bought by the patient

Through the PSP program, Roche will provide services like: Physiotherapy, Financial counselling, Psychosocial and Nutritional counselling to SMA patients.

"Given the majority of people with SMA in India remain untreated, we believe Evrysdi, with its highly efficacious clinical profile and oral administration advantage, will offer meaningful benefits for many living with this rare neurological disease," said Dr. Bruno Jolain, Chief Medical Officer, Roche Pharma India. "Administration of Evrysdi requires no hospitalization, no anesthesia, no specialized care center, no complex administration and no steroids. A simple oral administration gives SMA patients, treating physicians and caregivers more control over their daily lives."

"Throughout their lives, many people with SMA may lose their ability to perform critical movements, which can impact the ability to independently participate in different aspects of daily life and even be life altering. SMA patients in India have long lived without a viable treatment option. We are today encouraged by the availability of an approved solution in India," said Alpana Sharma, Co-Founder & Director Patient Advocacy at CureSMA Foundation of India.

"The launch of Evrysdi in India is an eagerly awaited milestone for our community. We appreciate Roche's commitment in making this drug available in India soon after its global launch and in developing a treatment that can be administered at home," said Archana Vashist Panda, Co-Founder & Director Patient Advocacy, CureSMA Foundation of India.

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