Proud Moment for Bharat: First room temperature drug for Sickle Cell Disease developed at 1 per cent of global price

In a significant move towards combating sickle cell anaemia, Prime Minister Narendra Modi initiated a national mission in July 2023 aimed at screening over crores of young individuals, including mostly the tribals. The objective of the mission was to detect the debilitating disease early and manage it with effective therapies.

The mission, outlined in the Union budget of 2023, placed emphasis on awareness campaigns and universal screening targeting individuals aged between 0 and 40 years in tribal regions spanning 278 districts across 17 states. With a visionary goal of eradicating the disease by 2047, the government set a comprehensive strategy in motion.

New Hope for Sickle Cell Disease Patients

A groundbreaking development in this endeavour has emerged with the creation of the first-ever stable room-temperature drug for sickle cell disease, named Hydroxyurea oral suspension. Remarkably, this indigenous innovation comes at a nominal price, less than one per cent of the global market price.

Announcing the breakthrough on social media platform X, the company responsible for the drug, Akums Drugs and Pharmaceuticals, attributed the achievement to the visionary leadership of Health Minister Shri Mansukh Mandviya and the invaluable guidance from esteemed Niti Aayog member Dr. V K Paul. The company highlighted the product’s significance, tailored specifically for patients across all age groups battling sickle cell disease, with thoughtful accompaniments like oral syringes for precise dosage administration.

In contrast to the steep import price of approximately Rs 77,000 for the global brand of Hydroxyurea solution, which necessitates storage at 2 – 8 degrees Celsius, Akums aims to make medication more accessible and affordable. In alignment with the objectives of the National Sickle Cell Anemia Mission outlined by Prime Minister Modi, Akums pledges to provide this life-changing medicine to the government at a cost of less than Rs 600, reflecting the spirit of “Make in India” and promising widespread benefit to the masses.

Union Health Minister, in response to this milestone achievement, extended congratulations to the company, hailing it as a significant step towards eradicating sickle cell disease. In a post on X, the minister expressed optimism about the impact of this medicine, particularly on tribal communities, echoing Prime Minister Modi’s vision to rid India of sickle cell disease.

The minister wrote, “Many congratulations to you for developing a medicine to prevent sickle cell disease. PM @NarendraModi ji had launched the Sickle Cell Anemia Eradication Mission by 2023. This medicine will prove to be a boon, especially for our tribal sisters, brothers and children and we will soon free India from sickle cell.”

Understanding the Prevalence and Impact

Reports shed light on the daunting reality faced by an estimated 20 million Indians battling sickle cell disease (SCD), a devastating genetic disorder. According to a 2023 report by DKMS BMST, a non-governmental organisation dedicated to combating blood cancer, India accounts for a significant portion of SCD cases globally.

Sickle cell disease, defined by the US Center for Disease Control and Prevention (CDC) as an inherited blood disorder, manifests when red blood cells, responsible for transporting oxygen throughout the body, assume a sickle-shaped form instead of their usual round shape. This abnormality severely impedes blood flow and oxygen delivery, causing a myriad of health complications.

Observed annually on June 19, World Sickle Cell Day serves as a reminder of the urgent need to raise awareness about this rare blood disorder, which significantly reduces life expectancy.

Children bear the brunt of this disease, with India ranking second globally in the number of SCD cases, a staggering 20 million. Distressingly, each year witnesses the birth of 150,000 to 200,000 children afflicted with SCD in India alone, facing a lifetime of chronic pain, anaemia, organ damage, susceptibility to infections, and a shorter life expectancy. Shockingly, 50-80 per cent of children diagnosed with SCD struggle to survive beyond the age of five.

Unlike healthy red blood cells that endure for approximately 120 days, sickle cells disintegrate within a mere 10 days, leading to severe anaemia and fatigue in patients. The absence of functioning red blood cells can also impede growth and development in children, resulting in delayed puberty and stunted growth.

The hallmark of SCD is excruciating pain, as sickle-shaped cells obstruct blood flow through various bodily tissues and organs, particularly the chest, abdomen, and joints. Additionally, these abnormal cells can damage the spleen and compromise the immune system, leaving patients vulnerable to a host of infections. Moreover, the eyesight of individuals with SCD is at risk, as sickle cells can obstruct blood vessels crucial for vision, potentially leading to blindness.

Complications of SCD extend to swelling in the hands and feet due to impaired circulation, further exacerbating the suffering endured by patients.

Until recently, there was no cure for SCD, with timely stem cell transplants representing the only hope for affected individuals. However, the development of a new medicine offers a glimmer of hope for patients grappling with this debilitating condition, signifying a significant breakthrough in the ongoing battle against sickle cell disease.

Prime Minister Narendra Modi’s Vision

Prime Minister Narendra Modi, addressing the pressing issue of sickle cell anaemia, emphasised the profound impact of the disease on families as he launched a comprehensive mission aimed at early detection and treatment. Speaking at an event in Madhya Pradesh, Prime Minister Modi underscored the importance of both screening and awareness strategies in combating the disease.

“The impact of a disease is not felt by the patient alone, but by the whole family. The mission combines both screening and awareness strategies to ensure early detection and treatment while making people aware that they suffer from such a disease, and may unintentionally transfer it to the next generation,” stated the Prime Minister.

The mission’s launch preceded assembly polls in Madhya Pradesh, Chhattisgarh, and Rajasthan states with a significant tribal population that accounts for a majority of sickle cell anaemia cases. With India bearing more than 50 per cent of global sickle cell anaemia cases, the disease emerged as a public health concern in central Indian states like Madhya Pradesh, Chhattisgarh, Odisha, Jharkhand, and Gujarat.

Expanding the scope of the mission, additional states targeted for intervention include Maharashtra, Rajasthan, West Bengal, Tamil Nadu, Telangana, Andhra Pradesh, Karnataka, Assam, Uttar Pradesh, Kerala, Bihar, and Uttarakhand.

The initiative was built upon a pilot project for screening launched two years before 2023 in Jhabua and Alirajpur districts, later extended to an additional 89 tribal blocks. This effort resulted in the examination of 9,93,114 individuals, with 18,866 identified with the sickle cell trait and 1506 diagnosed with the disease.

Drawing from a previous survey conducted by the Indian Council of Medical Research and Department of Biotechnology, screening encompassed 1,13,83,664 individuals across multiple states, revealing approximately 8.75 per cent (9,96,368) positive cases (trait: 949057, disease: 47311).

Highlighting the mission’s tangible impact, Prime Minister Modi distributed sickle cell genetic status cards to beneficiaries, stressing that such an initiative could potentially save the lives of 2.5 lakh affected children and families.

Made in India Medicines for Rare Diseases

It is essential for readers to recognise that this groundbreaking innovation to cure sickel cell disease marks another milestone for Bharat, adding to a series of achievements in indigenous medicine that have emerged in the past. Notably, Indian pharmaceutical companies have successfully produced medications for four rare diseases, significantly reducing the dependency on costly imported formulations, according to officials from the Union Health Ministry.

These advancements come as the ministry prioritises action related to 13 rare diseases, alongside sickle cell anaemia. Among the notable achievements are medications for Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet-Lennox Gastaut Syndrome, which have been approved and are now being manufactured domestically.

Additionally, officials reveal that four more drugs for three diseases are currently undergoing approval processes and are expected to be available by 2024. These include Tablet Sapropterin for Phenylketonuria, tab Sodium Phenyl Butyrate, tablet Carglumic Acid for Hyperammonemia, and Capsule Miglustat for Gaucher’s disease.

The cost reductions achieved through indigenous manufacturing are substantial. For example, the annual cost of the Nitisinone capsules, used in treating Tyrosinemia Type 1, is expected to decrease from Rs 2.2 crore to just Rs 2.5 lakh. Similarly, Eliglustat capsules, imported at a cost of Rs 1.8-3.6 crore per annum, will now be available domestically for Rs 3-6 lakh per annum.

The domestic manufacturing of drugs for Wilson’s disease, Dravet-Lennox Gastaut Syndrome, and sickle cell anaemia also promises significant cost savings for patients. The Trientine capsules, imported at Rs 2.2 crore per annum, will be available domestically for Rs 2.2 lakh, while Cannabidiol (oral solution), previously priced at Rs 7-34 lakh per annum, will now cost only 1-5 lakh per annum.

Furthermore, the commercial supply of Hydroxyurea Syrup, used in sickle cell anaemia treatment, is expected to commence by March 2024, with a tentative price of Rs 405 per bottle. This represents a considerable reduction from the current cost of 840 USD (Rs 70,000) per 100 ml for imported oral suspension.

The initiative to develop these drugs began in July 2022, with extensive consultations involving academia, pharmaceutical industries, organisations, CDSCO, and the Department of Pharmaceuticals. Subsequent interactions with drug manufacturers and the Drugs Controller General of India led to the approval of these drugs and the significant reduction in prices.

Rare diseases, affecting a small number of people, collectively impact 6-8 per cent of the population in any country at any given time. In India, officials estimate that there could be 8.4-10 crore cases, with nearly 80 per cent of these diseases being genetic in nature.