Globally, 60% of kids born with Spinal Muscular Atrophy (SMA) every year suffer from SMA Type 1. 68% of the children with type 1 SMA die before their second birthday and 82% of the children with type 1 SMA die before their 4th birthday.
In order to offer therapy for this rare disease, Roche recently announced the launch of Evrysdi® (risdiplam), the first and only approved treatment in India for SMA patients.
In an exclusive interview with Financial Express Online, V Simpson Emmanuel, CEO and Managing Director, Roche Pharma India talks about how the launch of Evrysdi has ushered a new journey of hope for patients living with SMA in India further explaining that Evrysdi is the first and only oral medicine and can be administered at home by mouth or a feeding tube once a day. Oral administration will empower patients to self-administer, have more control over their treatment regime and does not disrupt the patient’s daily routine.
Headquartered in Switzerland, Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system.
Can you share some data on the prevalence of SMA in India? What is the duration of the treatment required and how is it different?
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 live births globally and 1 in 7744 live births in India and is the leading genetic cause of infant mortality. To make things worse, SMA is one of leading genetic causes of death in children.
People with SMA have insufficient levels of Survival Motor Neuron (SMN) protein in the body which results in reduced function of nerves that control muscles and movement. Evrysdi addresses the underlying cause of SMA by increasing the production of the Survival Motor Neuron (SMN) protein. Evrysdi is a disease modifying therapy that has to be taken for life.
What role have existing patient and support programs played in addressing the cost and accessibility issue for SMA drugs?
Organisations like Cure SMA have been working tirelessly to create awareness around SMA and help with counselling parents with kids who are suffering this disease. We are also seeing increased efforts where patients are looking to raise funds in various crowd-funding platforms. We are highly encouraged by the government’s efforts in bringing out a Rare Disease policy. It is a great start and sets the tone for what can be achieved in the management of Rare Disease in the country.
Are there any compassionate use (CU) programs for SMA by Roche? Please share details – patients reached, approach and how do you contact these patients?
CU programs allow patients to access treatment where no other treatment options are available and where local regulations permit, before approval. We started our CU programs in India in December 2019. Our CU Program included patients who were either type 1 or type 2, were more than 2 months of age and had no access to any of the therapies. A total of 73 SMA patients spread across 27 public and private healthcare facilities have been included in the CU program. Till date, we have already provided over 600 bottles of Evrysdi under our India CU program.
Since this (Evrysdi) is an oral drug, and the first one in India, what does it means for SMA patients?
Evrysdi is the first and only approved treatment for SMA available in India. Oral administration empowers patients to self-administer, have more control over their treatment regime and does not disrupt the patient’s daily routine.
Evrysdi is designed to treat SMA by increasing production of the Survival Motor Neuron (SMN) protein. It starts working from Day1 of administration. The drug gets distributed to the entire CNS and throughout the body in a sustained manner. No other treatment has demonstrated that.
What kind of impact are you expecting on the accessibility of this drug?
With the launch of Evrysdi in India, patients now have access to an approved drug for SMA.
To further drive broader access to SMA patients in India, Roche is proud to announce its Patient Support Program for Evrysdi through which in the first two years of treatment, Roche will provide three bottles free for every two bottles bought by the patient. From the third year onwards, Roche will provide two bottles free for every one bottle bought by the patient
Evrysdi will be available through Roche’s distributor network across the country. For information one may call 1800-202-4755.
SMA patients have weak pulmonary health and find it difficult to visit a hospital to receive therapy, especially in COVID times. Moreover, motor disability adds to this burden and makes it very difficult for these patients and caregivers to travel to receive the drug. To solve this challenge, Roche will provide free home delivery of Evrysdi® to each and every patient following consent from the patient/ caregiver and their HCPs. Being the only oral medication for SMA that can be administered in a home setting, this facility will add tremendous convenience to both patients and their caregivers, especially the added challenge Covid-19 poses.
How does Roche plan to address the situation of rare diseases in India through this launch?
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.
The launch of Evrysdi in India is Roche’s commitment to living its purpose of doing now what patients need next. It is also a fine example of how we have partnered with stakeholders in the ecosystem to bring this product to market. We partnered with the entire SMA community; patients, physicians and regulators. The strength and resolve of the SMA community has continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them.
We have also partnered with 27 healthcare centers across the country through our CU Program. We have worked closely with different patient advocacy groups who have provided a tremendous service to the SMA community, especially the CureSMA foundation of India.
Going forward, we will also work collaboratively with health authorities, government agencies and other key stakeholders with the aim to ensure broad and rapid access to Evrysdi to all patients who can benefit from the treatment.