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FDA Orphan Drug Designation For Treatment Of Ebola Virus

Ebola Virus Particles






On August 25th, the FDA grants LeafBio Inc’s ZMapp product an Orphan Drug Designation (ODD) for the treatment of the Ebola virus infection:

FDA Orphan Product Designation Database Record

Generic Name: Monoclonal antibody consisting of three mouse/human chimeric IgG1 monoclonal antibodies (c2G4, c4G7, and c13C6) that target Ebola virus
Trade Name: n/a
Date Designated: 08-25-2014
Orphan Designation: Treatment of Ebola virus infection
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: LeafBio, Inc. 6160 Lusk Blvd., Suite C105 San Diego , CA 90121


LeafBio Inc., is the commercialization arm of Mapp Biopharmaceutical, a company developing novel pharmaceuticals for the prevention and treatment of infectious diseases. Mapp Biopharmaceutical focuses on unmet needs in global health and biodefense. As Mapp Biopharmaceutical’s products transition to clinical evaluation, LeafBio assumes ownership and commercialization responsibilities.

ZMapp  is the result of a collaboration between:

•   Mapp Biopharmaceutical (San Diego)

•   LeafBio (San Diego)

•   Defyrus Inc. (Canada)

•   U.S. government

•   Public Health Agency of Canada (PHAC).

ZMapp is made up of 3 humanized monoclonal antibodies manufactured in plants, specifically Nicotiana. It is an optimized cocktail that was first identified as a drug candidate in January 2014. ZMapp has not yet been evaluated for safety in humans and very little of the drug is currently available. Because ZMapp is an experimental product, only limited supplies were manufactured for testing in animals. ZMapp has shown efficacy in a monkey model of Ebola.

ZMapp was the experimental anti-viral drug given to American missionaries Kent Brantly and Nancy Writebol, as well as a Spanish priest and 3 African healthcare workers. One of the African healthcare workers died.

Mapp Pharmaceutical has only 9 employees. All of the samples of ZMapp are now gone and the company is looking for help from the federal government to make more. Meanwhile, the National Institutes of Health (NIH) has the go ahead to start testing a new Ebola vaccine in humans, with data maybe being available by the end of 2014. The vaccine is developed by NIH and Okairos, a biotech GlaxoSmithKline acquired in 2013.

Please Note: “Ebola Virus Particles” courtesy of NIH/NIAID [CC-BY-2.0 Generic].

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Lysosomal Storage Disorders: Gene Therapy And FDA Orphan Designations



No gene therapy has ever been approved for sale in the United States. In Europe, the EMA (European Medicines Agency), has given regulatory approval to UniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD).

This is the twelfth Blog Post in a continuing series that examines Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post identifies gene therapies for LSDs that have received FDA Orphan Drug Designation in 2013 – 2014.

FDA Orphan Designated Gene Therapies For LSDs (2013 – 2014) 

Drug Name Sponsor Company Indication FDA ODD Date
AAV-G6Pase vector GlyGenix Therapeutics Glycogen storage disease type Ia 03.11.13
Recombinant adenovirus vector AAV2/rh8 expressing human B-hexosaminidase A & B subunits National Tay-Sachs & Allied Diseases Association (NTSAD) Tay-Sachs Disease 03.25.13
Recombinant adeno- associated virus vector AAV2/rh8 expressing human B-hexosaminidase A and B subunits National Tay-Sachs & Allied Diseases Association (NTASD) Sandhoff Disease 03.25.13
Adeno associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs Lysogene (France) MPS III A (Sanfilippo Syndrome) 05.06.13
Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase AbeonaTherapeutics MPS III A (Sanfilippo Syndrome) 04.29.14
Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase Abeona Therapeutics MPS III B (Sanfilippo Syndrome) 04.30.14


Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons..

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

FDA Breakthrough Therapy Designation: Statistics Chart Updated (as of 08/22/14)

FDA logo

The CBER BTD #s are current as of 07/31/14 and the CDER BTD #s are current as of 08/22/14.

The changes are as follows :

•   The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 207 to 215

•   The total # of FDA CDER + CBER BTDs Granted by the FDA increases from 58 to 61

•   The total # of FDA CDER + CBER BTDs Denied by the FDA increases from 113 to 117

•   The total # of FDA CDER + CBER BTDs Pending by the FDA increases from 36 to 37.

FDA CBER + CDER BTDs as of 08/22/14

Breakthrough Therapy  Designation (BTD) Category Total # of CBER Designations (07/09/12-07/31/14) Total # of CDER Designations (07/09/12-08/22/14) Total # of CBER +   CDER BTD Designations (07/09/12-08/22/14)
Total # of BTD Requests Received 32 183 215
Total # of BTDs Granted 6 55 61
Total # of BTDs   Denied 25 92 117
Total # of BTDs   Pending 1 36 37



•   28.4 %    of the total # of BTD Requests Received results in the BTD being granted

•   54.4 % of the total # of BTD Requests Received results in the BTD being denied

•   17.2 % of the total # of BTD Requests Received results in the BTD pending.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Orphan Drugs At BioPharma Mexico 2014

BioPharma Mexico banner250x250

The 2nd Annual BioPharma Mexico conference, November 4 – 5, 2014, in Mexico City, brings together COFEPRIS, payers, government, biopharma, academia, and other stakeholders to talk about strategies to bring biocomparables, stem cells, and orphan drugs to market faster.

No orphan drugs are currently manufactured in Mexico nonetheless, orphan diseases have gained more recognition in the health and pharma communities. Despite the limited number of patients for a particular drug, this market is on the rise in Mexico.

Victor M. Anaya Bourgoing, General Director of Genzyme Mexico and Raul Vivar, Country Medical Head of Shire Mexico, will both be speaking on the state of orphan drugs at this year’s BioPharma Mexico.

Topics on orphan drugs in Mexico to be discussed include:

•   Understanding where COFEPRIS stands on regulation

•   Do’s and don’ts of importing orphan drugs

•   Accessing rare disease patients

•   Recent Orphan Drug Act in Mexico

•   Raising awareness of orphan diseases.

A compiled list of the top 20 trends in the Mexican pharma industry, from regulation to market access, is available as an eBook to download. The eBook includes segments on orphan drugs, patient groups, and awareness of orphan diseases.

Download the eBook.

To join or find out more about the event, visit the event website.

Meeting Logo courtesy of BioPharma Mexico 2014. The Orphan Druganaut Blog is a Media Partner.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

FDA Breakthrough Therapy Designation: GSK Receives Approval For Additional Indication For Promacta


GlaxoSmithKline (GSK) announces on August 26th that the  FDA approves a supplemental New Drug Application (sNDA), for the once-daily use of Promacta (Eltrombopag) in patients with Severe Aplastic Anemia (SAA), who have had an insufficient response to Immunosuppressive Therapy (IST). Promacta is a new 1st-in-class treatment option for previously treated SAA patients.

Eltrombopag is marketed under the brand name Promacta in the US and Revolade in most ex-US countries. In addition to the approval of Promacta for SAA in the US, eltrombopag is indicated for the treatment of thrombocytopenia in patients.

SAA is a rare disorder where the bone marrow fails to make enough new blood cells. There are currently no therapies approved for this indication. About forty percent (40%) of patients who do not respond to initial IST die within 5 years of diagnosis.

FDA Regulatory Actions

•   Orphan Drug Designation (ODD) in May 2008 & approval in November 2008 for Thrombocytopenia

•   ODD in November 2013 for Aplastic Anemia

•   Breakthrough Therapy Designation (BTD) in February 2014 for cytopenias in patients with SAA who have had insufficient response to IST

•   Priority Review in April 2014

•   Approval for additional indication in August 2014 for patients with SAA who have had insufficient response to IST.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Guest Blogger: Findacure – Understanding The Common Through The “Rare”

Findacure logo 260x200

The Orphan Druganaut Blog is honored to have Rhys Dore, an Oxford University medical sciences student and a Findacure volunteer, as a Guest Blogger. Findacure is a charity which focuses around fundamental diseases – rare, often genetic, diseases from which the basic mechanisms of dysfunction can be extrapolated and applied to our understanding of more common conditions.

Rare diseases are the magnifying glass. They magnify and expose the importance of affected genes. The necessities of the disrupted physiological mechanisms are shown by the frequently severe symptoms which are found in such patients. These mechanisms are not solely affected in rare diseases; they are often altered in more common conditions. Thus these rare diseases can be regarded as fundamental to understanding the foundations of illness.

For example, take a rare deficiency in CD73 enzyme. This results in increased levels of a molecule called adenosine, leading to abnormal calcium deposits in the arteries and joints. This type of deposits parallels that found in cardiovascular disease and certain bone abnormalities. This discovery has paved the way for further investigations into the role of adenosine in these conditions. This is but one of many insights which are found to cross the boundary between fundamental and common diseases. Many fundamental diseases additionally have similar pathological parallels which are often more extreme and earlier-presenting. An extreme example of this is the group of progeroid syndromes which are characterised by premature ageing. Studying these syndromes not only provide a useful model upon which to analyze the detriments of ageing, but certain forms reveal the process of ageing in specific organs.

It is also beneficial to assess the differences found between similar fundamental diseases and their subtypes. Conditions such as Bardet-Biedl Syndrome and Alström Syndrome are similar in pathology; both are due to defective cell-signalling components called cilia and result in problems such as sensory defects and obesity. However, patients diagnosed with Alström Syndrome are significantly more likely to develop type II diabetes. Therefore studying what differs between these syndromes may reveal a contributing factor to the development of hereditary diabetes. These key differences are frequent due to fundamental diseases typically presenting as a collection of various pathologies rather than one key identifiable dysfunction.

Many fundamental diseases have proven vital for the basics of research investigations. This is particularly important for research based upon extreme animal models where the basic underlying mechanisms have a reasonable level of similarity to human physiology. Immunological research heavily depends on this system. One mouse model has been found to be reasonably similar to the human autoimmune condition APS type-1; both lack the Aire gene. This has allowed extensive research on the disease’s interaction with inflammatory messengers and antibodies. Such results too provide information concerning general autoimmunity and may be applied to a range of common diseases that have been associated with Aire-mechanism dysfunctions such as systemic sclerosis, alopecia, vitiligo, and even type I diabetes.

Furthermore, it should be noted that basic mechanism elucidation is only the first step of research. There is much potential for the repositioning of drug treatments from fundamental to common diseases. This is shown in hereditary hyperhomocysteinaemia (HHH), a disease of excess homocysteine resulting in increased cardiovascular disease. The original therapy for HHH involves using B-vitamins to reduce homocysteine levels, which reduces cardiovascular complications and has the potential for preventing cerebral infarctions otherwise. Interestingly, this B-vitamin treatment is also currently being investigated for its preventative effect against Alzheimer’s. Drug repositioning leads to huge cost reductions as drug treatments will often already be in production and be optimised for safety.

The altruism of fundamental disease patients to partake in clinical research is itself essential for the advanced progression of many therapies. As highlighted in Gorlin syndrome research; a disease characterised by excessively frequent basal cell carcinomas starting in adolescence. These are benign skin cancers which were found to result from an overactivation of the ‘Hedgehog pathway’. An inhibitor of this pathway, vismodegib, was discovered and tested using Gorlin Syndrome subjects as their high incidence of skin cancers allows more extensive data to be gained. These studies were found to be successful and vismodegib has since gained FDA approval for the use of such cancers in Gorlin Syndrome and otherwise; a particularly wide-reaching achievement as basal cell carcinomas are the most common form of skin cancer worldwide.

So perhaps the magnifying glass analogy is not quite correct. Fundamental diseases do not simply highlight the problems; they provide a gateway through which research and innovation can be applied to common disease. Fundamental diseases are the looking glass, leading to a so-called ‘Wonderland’ of heightened understanding and novel therapies applicable to both rare and common conditions alike. It is this revelation that Findacure: The Fundamental Disease Partnership ( aims to promote and explore via collaborating with patients, researchers and pharmaceutical industry to guide us into an era of appreciation of the fundamental diseases.

Logo courtesy of Findacure.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Rare Diseases And Clinical Trials: Interview With Dr. Nick Sireau

Nick Sireau interview image

As the 5th World Orphan Drug Congress is soon approaching this November, from 12-14 in Brussels, we sat down with Nick Sireau, Chairman and CEO of the AKU Society, and one of our key speakers at the Congress for a short interview.

Dr. Sireau will be addressing the importance of having an all-stakeholder perspective of clinical trials for the rare disease AKU (Black Bone Disease). Dr. Sireau is also the Co-founder and Chairman of Findacure, a new social enterprise that raises awareness into, and helps patients with, extreme and exceptional diseases that advance understanding of medicine and help discover potential new treatments. As a committee member of Rare Disease UK, the national alliance of rare disease groups in the UK and of the International Rare Disease Research Consortium (IRDiRC), Dr. Sireau has extensive experience in putting together rare and ultra-rare clinical trials.

What is the importance of having an all-stakeholder perspective of clinical trials, addressed in your talk?

Clinical trials increasingly need an all-stakeholder approach in order to be successful. Our experience with DevelopAKUre, a consortium developing the drug nitisinone for the rare disease AKU (Black Bone Disease), shows that bringing together clinicians, academics, industry and patients on equal terms is an excellent way of overcoming the challenges involved in rare disease trials. Each party brings its own perspective, resources and skills to the consortium – allowing us to develop innovative ways of devising and implementing the trials.

What are some of the most pressing issues you are facing in implementing this?

As is often the case, a key challenge is funding. Despite the €6 million from the European Commission (EC) and the in-kind contributions of consortium members, we are pushing our resources to the limit. On the one hand, this has forced us to be even more creative in our approach. On the other hand, it shows how difficult it is to carry out public-private partnerships in areas of market failure such as in many rare diseases. Without funding from the EC, it would be difficult for many rare disease groups to work on new treatments for their disease. That’s why we need a variety of funding avenues, not just from international sources such as the EC, but increasingly from national and philanthropic sources.

What do you think will be the single biggest trend or change to the orphan and rare disease sector between now and 2025?

I hope the biggest change in the orphan and rare disease sector over the next 10 years will be a surge in patient involvement in clinical trials. Patient groups have a passion and clarity of vision that can bring together all the other actors in the process by focusing on the need to develop cures fast and effectively. They are also increasingly skilled in their approach, thanks to the training and support provided by umbrella groups such as EURORDIS. If this continues, I hope we will see more projects where patients are leading the clinical development of treatments.

What would you like to gain from our congress?

The World Orphan Drug Congress is a fantastic opportunity for finding out about the latest developments in the field of orphan drugs and rare diseases. It’s also an excellent networking opportunity. I’ve made a number of very good contacts there in the past that have led to some of the key collaborations that are the center of our DevelopAKUre consortium. I hope to gain similar knowledge and contacts at this year’s congress.

To hear more please visit our website to book your place so you can meet other key speakers like Nick Sireau as they share solutions for collaborative orphan drug clinical trials.

Photo courtesy of NickSireau/AKU Society/Terrapinn. The Orphan Druganaut Blog is a Media Partner.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.


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