MedNess is back with big news from the pharma companies. CAR-T therapy is getting closer to the market and sickle cell anemia sees a new yet ‘not-so-new’ therapeutic development. Stock prices of Indian pharma giant Biocon soar to a record high with a recommendation for a biosimilar that can be used for treating breast cancer. Do not miss out on the details of what these companies have in mind, and how are their competitors faring.
MedNess North America
Novartis poised to win CAR-T race
In a historic move on July 12, the US Food and Drug Administration (FDA)’s Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of Novartis’ CTL019 (tisagenlecleucel-T), a groundbreaking CAR-T therapy for treating relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) in children and young adults. This decision is expected to lead to approval of the therapy by US FDA on or before October 3.
— Sunil Joshi (@pavansut) July 12, 2017
Due to the complexity of the therapy and expert care needed to manage side-effects, Novartis upon approval will restrict CTL019 use to 30-35 treatment centers in the U.S. who will be trained on a comprehensive risk-mitigation strategy that drew high praise from several ODAC members during the hearing. Novartis has plans for additional filings for CTL019 later this year, including applications with the FDA and European Medicines Agency (EMA) for the treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL).
Close rival Kite Pharma’s CAR-T application for the treatment of adults with advanced and aggressive lymphoma will receive a decision from FDA in November. Other key players being keenly watched in this field are Juno Therapeutics, Bluebird Bio, and Cellectis.
The announcement, however, did not affect Novartis’ or Oxford BioMedica’s shares, the companies supplying raw materials for CTL019. Before this announcement, Oxford BioMedica renewed its contract with Novartis extending its partnership for the commercial and clinical supply of its LentiVector gene delivery technology. On the other hand, Kite Pharma shares have seen big swings and is currently trading 24% higher than Novartis.
FDA approves first sickle cell disease treatment in 20 years
On July 7, the US FDA approved Endari for patients with Sickle Cell Disease (SCD) to reduce severe complications associated with the disorder. Endari, developed by Emmaus Lifesciences Inc. based in Torrance, CA, is the first FDA-approved treatment for pediatric patients with SCD and the first new treatment in almost 20 years for adult patients. The therapy is an orally administered pharmaceutical grade L-glutamine. The therapy’s efficacy and safety were evaluated in a 48-week Phase 3 trial (NCT01179217) involving sickle cell patients between the ages of 5 and 58 who had two or more painful crises in 12 months prior to enrollment. Results from the trial demonstrated that Endari reduced the frequency of sickle cell crises by 25 percent and hospitalizations by 33 percent.
The FDA approved Endari, the first new drug in nearly 20 years to treat sickle cell anemia complications. https://t.co/befZyEGt3q
— Ashton Tweed (@AshtonTweedLtd) July 11, 2017
Sickle cell disease is a rare, inherited disorder characterized by abnormally sickle-shaped red blood cells. These malformed red blood cells clog blood vessels and cut off oxygen to the body’s tissues, leading to episodes of severe pain and organ damage. Approximately 100,000 people in the U.S. have sickle cell disease, according to the National Institutes of Health. Endari helps increasing free glutamine in blood, which is taken up by the sickle red blood cells and used to generate anti-oxidant molecules as a product of glutamine degradation. These new antioxidants help neutralize the oxidative stress in sickle cells, allowing them to regain the flexibility needed to travel through blood vessels and capillaries, carrying oxygen to tissues.
L-glutamine, the active ingredient in Endari can be purchased over the counter, which could complicate Emmaus’s ability to obtain insurance coverage. Ontario-based Generex Biotechnology acquired controlling interest in Emmaus LifeSciences in March 2017. The recent FDA approval saw an 8% increase in share value for Generex Biotechnology.
Biocon flying high with US FDA recommendation
Biocon’s biosimilar of Trastuzumab, a breast cancer drug obtained unanimous recommendation (16-0) from US FDA ODAC – taking the drug closer to procuring US FDA’s marketing approval. Trastuzumab, originally developed by Roche is commonly used to treat HER2-positive breast cancer and has a multi billion-dollar market globally with an estimated market of $6.9 bn in the US alone. Biocon chairperson Kiran Mazumdar-Shaw said that they expect to obtain approval to launch their product by September 3.
— Biocon (@Bioconlimited) July 14, 2017
Biocon collaborated with US drug maker Mylan to develop this biosimilar. Biocon will manufacture the product for all markets globally with a profit sharing arrangement with Mylan for its markets. However, with numerous observations from US FDA and European regulators, Biocon faces concerns regarding quality lapses. To circumvent these Ms. Mazumdar-Shaw mentioned that the company has GMP certification for its drug substance facility for trastuzumab and will expedite the process to seek an early re-inspection.
It is important that the approvals work swiftly for Biocon due to the risk of losing market share to other generics manufacturing competitors . One company to watch out for will be Aurobindo Pharma – the newest entrant in oncology segment in India. It plans to file for 15-17 products in oncology this year and expects to be able to start selling by end of the year. Last month, Dr. Reddy’s Laboratories and its partner Natco Pharma obtained US FDA approval for the generic version of Doxil, a chemotherapy drug used for ovarian cancer, Kaposi’s sarcoma, etc. to launch in the US market.
While the US FDA approval recommendation has already caused a surge of 11.8% in the stock prices of Biocon, it will need to concentrate highly on strengthening its technical capabilities and avoid quality lapses. It cannot afford to lose time in getting the approvals in order to avoid losing the first mover’s advantage in the market. Aurobindo Pharma has already seen a surge of 22.8% since it announced plans to file its oncology products.
About the authors:
Radhika Gopal is currently a postdoctoral research associate at The Scripps Research Institute, La Jolla, where she works on Hepatitis C virus glycoproteins. She also chairs the Corporate Sponsorship Committee for the San Diego chapter of AWIS (Association for Women in Science). Radhika has completed project management and business fundamentals courses from UCSD, and is actively seeking opportunities in the Biotech Industry. In her spare time, she enjoys music, hiking, reading music and the beach. She can be reached here.
Somdatta Karak, PhD is interested in pharma and healthcare sector in Asia. She also works with PhD Career Support Group / Club SciWri as its project coordinator. She aims to make a more and better informed world for all, and hence experiments with making effective platforms of education. She can be reached here.
About the editor:
Sayantan Chakraborty is an IRTA postdoctoral visiting fellow at the National Institute on Aging – National Institutes of Health, Baltimore, USA. Apart from science, he invests his time in networking, writing, and organizing events. He is consolidating his efforts to build a platform that brings together scientists and industry professionals and to help spread the perception of alternate careers for life science graduates.
Follow him on Twitter @ch_sayantan
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