The Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval of DARZALEX FASPRO™ (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, for the treatment of patients with light chain (AL) amyloidosis, a rare and potentially fatal disease for which there are no currently approved therapies. The sBLA is supported by positive results from the Phase 3 ANDROMEDA study, which were presented as a late-breaking abstract at the 25th European Hematology Association Annual Congress in June. ANDROMEDA evaluated subcutaneous daratumumab in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) compared to VCd alone and met its primary endpoint of overall hematologic complete response rate. 

"We are excited about the potential of helping patients with AL amyloidosis who currently have no FDA-approved therapies for the treatment of their disease," said Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology, Janssen Research & Development, LLC. "The results from the Phase 3 ANDROMEDA study also provide preliminary evidence of DARZALEX FASPRO's potential to modify the organ damage that is a hallmark of this serious disease with high unmet needs and we look forward to collaborating with the agency in the review of the application."

The sBLA is being reviewed under the FDA Real-Time Oncology Review (RTOR) program, which allows data for certain applications to be reviewed before the applicant formally submits the complete application. The RTOR program aims to explore a more efficient review process to help ensure treatments are available as soon as possible for patients. Selection into the RTOR program does not guarantee or influence approvability of the supplemental application.

The submission is also being reviewed under Project Orbis, an initiative of the FDA Oncology Center of Excellence, which provides a framework for concurrent submission and review of oncology medicine applications among international regulatory agencies.

AL amyloidosis is a life-threatening disorder that occurs when plasma cells in the bone marrow produce abnormal light chains, that form amyloid deposits, which build up in vital organs and eventually cause organ deterioration. The disease can affect different organs in different people, but the most frequently affected organs are the heart, kidneys, liver, spleen, GI tract and nervous system. Diagnosis of AL amyloidosis is often delayed because patients present with non-specific symptoms that mimic other conditions, resulting in a poor prognosis. There are currently no FDA-approved therapies to treat this devastating disease. While AL amyloidosis is the most common type of amyloidosis, it remains a rare disease with an estimated 30,000 to 45,000 people living with the disease in the U.S. and Europe. Each year, an estimated 4,500 people develop AL amyloidosis in the U.S. alone.

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Doctors at the Krishna Institute of Medical Sciences (KIMS), one of India’s leading healthcare providers, have successfully performed India’s first-ever double lung transplant surgery on a Covid-19+ve patient, who was discharged from the hospital on Friday. The procedure was performed at the main branch of the hospital located at Hyderabad, and was led by Dr. Sandeep Attawar, who is considered a pioneer in heart and lung transplant surgeries in India.

The patient, a Rizwan (Monu) 32-year-old man from Chandigarh, Punjab, was suffering from sarcoidosis that affected his lungs significantly, leading to fibrosis of the lungs. Condition of the patient deteriorated rapidly. A double lung transplant was the only permanent option for cure. The situation worsened when the patient contracted coronavirus while awaiting for his double lung transplant. This worsened matters as it increased his oxygen requirement from 15 liters/minute to 50 liters/min for the past 8 weeks.

Commenting on the procedure, Dr. Sandeep Attawar, Thoracic Organ Transplantation Surgeon, KIMS Heart & Lung Transplant Institute, KIMS Hospitals said, “The patient was a severe case of lung sarcoidosis, and Covid-19 virus only complicated the precarious lung condition. Fortunately, a match to his lungs was found in a person declared braindead in Kolkata, and the harvested lungs were airlifted to Hyderabad to save the life of the patient. The procedure was complex and had little scope for errors; and only a timely transplant of lungs helped save the patient. What is more important is that good outcomes are difficult to achieve as these patients are extremely sick, under nourished and bed ridden before the transplant. Post discharge, he will need close monitoring, bio bubble environment & careful drug control for 6 weeks at least.

Dr. Sandeep Attawar, is the most experienced heart and lung transplant surgeons in the country. A veteran in the field of transplant surgeries with over 24-years’ experience, Dr. Attawar has to date performed over 12,000 heart surgeries, and has over 250 transplant surgeries for lungs, heart, and artificial heart implants (LVAD) to his credit.

The prevailing scenario around the world in general and India is compelling patients to stay indoors for a prolonged period. While people are ignoring/neglecting certain ailments and not seeking medical care at the right time, prolonged neglect could result into complex ailments. Hence, it is suggested that people stay aware of the symptoms of possible ailments they could contract and respond to it with medical care.