Skip to content

FDA Breakthrough Therapy: TaiMed Biologics’ HIV And Atara Biotherapeutics’ T-Cell Therapy


The FDA recently grants 2 FDA Breakthrough Therapy Designations (BTDs):

•   TaiMed Biologics’ Ibalizumab for HIV/AIDS

•   Memorial Sloan Kettering Cancer Center (MSKCC) and Atara Biotherapeutics’ EBV-CTL, T-Cell Therapy for complications tied to bone marrow transplants.

I – TaiMed Biologics’ Ibalizumab For HIV/AIDS

TaiMed Biologics, a Chinese biotechnology company, receives last week, the FDA BTD for Ibalizumab (TMB-355) for the treatment of HIV/AIDS. WuXi PharmaTech, TaiMed Biologics’ Shanghai-based manufacturing partner, plans on filing a rolling FDA Biologics License Application (BLA). The FDA approved Ibalizumab in May 2014 for the treatment of patients on expanded access.

This is the first Chinese drug to receive a FDA BTD. No Asian company has received a FDA BTD, except for Japan’s Takeda Pharmaceutical, which receives in December 2014,  a FDA BTD for Ixazomib, for the treatment of relapsed or refractory systemic light-chain Amyloidosis (AL).

Ibalizumab is a humanized monoclonal antibody (mAb) and part of an emerging class of HIV therapies known as viral-entry inhibitors. The drug works by binding to the CD4 molecule, the primary receptor for HIV infection, thereby interfering with the penetration of the virus into the cell. It is the first entry-blocking humanized mAb to treat HIV/AIDS. If Ibalizumab is approved by the FDA, it will be the first biologic product manufactured in China to be launched in the US market.

II – MSKCC And Atara Biotherapeutics T-Cell Therapy

On March 2nd, Atara Biotherapeutics and partner MSKCC, announce that the FDA BTD is granted to Atara Biotherapeutics’ optioned cytotoxic T lymphocytes activated against Epstein-Barr Virus (EBV-CTL). This is for the treatment of rituximab-refractory, EBV-associated lymphoproliferative disease (EBV-LPD), a type of malignancy occurring after allogeneic hematopoietic cell transplantation (HCT). Allogeneic HCT is also commonly called a bone marrow transplant.

In September 2014, Atara Biotherapeutics, a San Francisco-based company, forms an alliance with MSKCC for developing and commercializing allogeneic T-Cell therapies for the treatment of certain cancers and viral infections. Atara Biotherapeutics executes a $55 million IPO in October 2014.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “White Pocket Reflection” by John Fowler (Flickr: White Pocket Reflection) [CC BY 2.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

FDA February 2015 Products Receiving Orphan Designation






The chart below identifies FDA February 2015 Products Receiving Orphan Designation as of 03/01/15 in ascending “Orphan Drug Designation Date” order.

FDA February 2015 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1  N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide/ 02.03 Ignyta Inc. TrkA+, TrkB+, TrkC+, ROS1+ & ALK+ NSCLC
2 Antinuclear antibody conjugated liposomal doxorubicin/ 02.03 NanoSmart Pharmaceuticals Ewing’s Sarcoma
3 Bortezomib/ 02.03 Millennium Pharmaceutials Acute lymphoblastic leukemia
4 Saposin C/ 02.03 Bexion Pharmaceuticals Glioblastoma multiforme
5 Tisagenlecleucel-T/ 02.03 Novartis Diffuse large B-cell lymphoma
6 Copanlisib/ 02.05 Bayer HealthCare Follicular lymphoma
7 Ibrutinib/ 02.05 Pharmacyclics Splenic marginal zone lymphoma
8 Ibrutinib/ 02.05 Pharmacyclics Nodal marginal zone lymphoma
9 Tolerogen/ 02.05 Toleranzia AB (Switzerland) Myasthenia Gravis
10 Omeprazole-lansoprazoe with buffer/ 02.10 Effexus Pharmaceuticals Esophageal ulcers
11 Pelareorep/ 02.10 Oncolytics Biotech (Canada) Ovarian Cancer
12 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6yl]-2-pyrimidinamine/ 02.10 Verastem Malignant mesothelioma
13 Levomefolate calcium/ 02.10 Cox Biosciences Megaloblastic anemia caused by folate deficiency
14 Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate/ 02.10 Glaxo Group Limited (UK) AL amyloidosis
15 Recombinant monoclonal antibody to human serum amyloid P component/ 02.10 Glaxo Group Limited (UK) AL amyloidosis
16 Paromomycin/ 02.11 The Surgeon General, Dept. of the Army Cutaneous leishmaniasis
17 3-pentylbenzenacetic acid sodium salt/ 02.11 ProMetic Life Sciences (Canada) Idiopathic pulmonary fibrosis
18 Glycyl-L-2-methylpropyl-L-glutamic acid / 02.11 Neuren Pharmaceuticals (New Zealand) Rett syndrome
19 Bivalent anti-human myostatin adnectin-IgG1/ 02.11 Bristol-Myers Squibb Duchenne muscular dystrophy
20 Recombinant humanized anti-interleukin 13 (IL-13) monoclonal antibody / 02.11 Receptos Inc. Eosinophilic esophagitis
21 Pelareorep/ 02.11 Oncolytics Biotech (Canada) Pancreatic Cancer
22 N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide/ 02.12 Ignyta Inc. TrkA+, TrkB+, TrkC+, ROS1+ & ALK+ Colorectal Cancer
23 Defactinib/ 02.12 Verastem Ovarian Cancer
24 Bovine Lactoferrin/ 02.19 Metrodora Therapeutics Late-onset sepsis in very low birth weight infants
25 Polidocanol/ 02.19 Provensis Ltd (UK) Congenital venous malformations
26 Naltrexone/ 02.23 Allodynic Therapeutics Postherpetic neuralgia
27 Andexanet Alfa/ 02.23 Portola Pharmaceuticals Reversing the anticoagulant effect of direct or indirect factor Xa inhibitors
28 Bovine Lactoferrin/ 02.23 Metrodora Therapeutics Prevention of necrotizing enterocolitis in very low birth weight infants
29 Rituximab/ 02.23 Genentech Pemphigus Vulgaris
30 Pelareorep/ 02.24 Oncolytics Biotech (Canada) Fallopian tube cancer
31 Deferasirox/ 02.24 Novartis Chronic iron overload in alpha-thalassemia
32 Acetylcysteine effervescent tablets for oral solution / 02.24 Arbor Pharmaceuticals Hepatic injury from acetaminophin overdose
33 Propranolol hydrochloride and etodolac / 02.24 Vicus Therapeutics Hepatocellular Carcinoma
34 Ketotifen/ 02.25 Melbourne Laboratories Mastocytosis


** Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

Please Note : “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-30] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

In Celebration Of Rare Disease Day: The Amish And Mennonites


In Strasburg, in the heart of Pennsylvania’s Amish country, is the Clinic for Special Children (CSC), a gene research clinic specializing in genetic problems of the “Plain People”, such as the Amish and the Old Order Mennonites. CSC is a nonprofit medical facility for children with genetic disorders and complex medical needs. CSC serves over 2,500 patients from 34 states and 17 countries, representing 150 unique genetic disorders.

CSC is founded in 1989 by Dr. D. Holmes Morton, a Harvard-trained pediatrician specializing in biochemical genetics, and his wife Caroline, after diagnosing a young Amish boy with Glutaric Aciduria Type 1 (GA1). GA1 is a metabolic disease that had previously been identified in only 8 other patients. GA1 is one of about 150 diseases or genetic mutations CSC has identified that “disproportionately affect the area’s Amish and Mennonites – a community of 60,000 descended from fewer than 100 settlers who came to Pennsylvania in the 1700s.” This has resulted in a limited genetic pool and the population is at high-risk for certain inherited genetic disorders, such as Maple Syrup Urine Disease and GA1. Per Dr. Holmes Morton:

“Special children are not just interesting medical problems, subjects of grants and research. Nor should they be called burdens to their families and communities. They are children who need our help, and if we allow them to, they will teach us compassion. They are children who need our help, and if we allow them to, they will teach us love. If we come to know these children as we should, they will make us better scientists, better physicians, and thoughtful people.”   


25 Years of Personalized, Genomic Medicine in Lancaster County

Washington Post (10.21.14) article, “Pennsylvania clinic treats genetic disorders in Amish and Mennonite people”.

Please Note:The Amish at Lancaster (5527842029)” by Serge Melki from Indianapolis, USA (The Amish at Lancaster uploaded by russavia) [CC BY 2.0] | via Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

In Celebration Of Rare Disease Day: The Human Side Of Epidermolysis Bullosa


In celebration of Rare Disease Day, the following is a republication of an interview that the Orphan Druganaut Blog was honored to have with Michaela O’Brien, director and producer of “In Crystal Skin”, a documentary on the human side of what it is like to live with the rare disease Epidermolysis Bullosa (EB) in a South American country. In 2014, a successful Indiegogo campaign is completed raising $25,000 to complete the film’s post-production.

EB is a rare genetic connective tissue condition that involves very fragile skin that blisters or tears with the slightest trauma or friction. EB patients are known as having “Crystal Skin”, and are also referred to as “Butterfly Children” due to the fragile nature of the skin, similar to the fragility of the wings of a butterfly. There is currently no cure or effective treatment, outside of daily wound care, pain management, and preventative bandaging.

The biopharmaceutical company, Scioderm, announces in April 2013that the company’s investigational topical cream, SD-101, receives the FDA’s Breakthrough Therapy Designation for the treatment of patients with inherited Epidermolysis Bullosa (EB). Since 2014, 4 products have received FDA Orphan Drug Designation (ODD) for EB.

Please tell us a little about your relationship with the EB patients that you followed in Colombia ?           

The first EB patient I met was in an orphanage in Bogotá, Colombia. She caught my eye because her body was almost completely covered in saran wrap and I wanted to understand why. I had never heard of EB before. She was courageous enough to share her story with me and introduced me to her sister, who also has EB. Through these two brave women, I was introduced to many other EB patients. One man is the oldest known patient living with EB in Colombia at 43 years old. Another is a 12-year-old girl, whose story sheds light on the family dynamics of managing life with a rare disease. 

What is it like from the patient perspective to live with a rare disease that currently has no cure ? What is the impact on the families or caretakers ?

Living with a rare disease which currently has no cure is tremendously difficult both for the patient and their family or caretaker. There is no end in sight for their disease or for the difficulties it causes. It can be a struggle to muster hope for the future when science can offer patients no resolution. EB is a disease of constant management, it requires intense care and maintenance, so though there is no cure, patients with EB hope for products and orphan drugs to alleviate their wounds and lessen the routine of constant care.   

What does Colombia offer from both a social, medical, and financial perspective to help those living with EB and rare diseases ? 

DEBRA or the Dystrophic Epidermolysis Research Association is an international organization dedicated to funding research and providing services to those with EB. It functions in many countries around the world, but some have existed for decades while others for only a few years, and some have more financial resources from their countries and governments. DEBRA Colombia is a very small organization, spearheaded by a few dedicated professionals who are very caring and supportive, but it is strapped for internal resources and medical professionals and products to care for their patients. From what I saw there seemed to be no other source for social, medical, or financial care for EB specifically. There is also an issue of general rare disease awareness and knowledge in Colombia, as well as many other countries. For example, a medical professional in Colombia explained to us that they only have 43 registered EB patients, and they estimated that given the population of the country there is about 90% of the EB patients left to be diagnosed and treated.   

What have you learned from making “In Crystal Skin” and following these patients in their everyday lives ? 

While making “In Crystal Skin” I have learned a great deal about hope and perseverance from patients. I am continually impressed with patient’s ability to remain positive in the face of adversity. The people I have met with rare diseases want to move forward in life just like anyone else, and they have dreams and aspirations which they live to fulfill. One of the most beautiful forms of intelligence that those with rare diseases have is the understanding of uniqueness, and how to be proud of it. EB and rare disease patients desire to understand and express their individuality and deserve that utmost respect for that courageous act.     

Is there a particular moment or conversation that affected you and stands out as a message to others about living with a rare disease ?

There are many moments from filming in Bogotá, Colombia with the subjects of “In Crystal Skin” which stand out in my mind. One particular conversation was with a young mother named Jackeline, whose 12 year old daughter has dystrophic EB. One day while filming she broke down in tears and expressed very candidly the difficulties of being a mother of a child with a rare disease–the limitations it puts on a young woman basically confined to the house in order to provide constant care for her child, the financial burdens it places on a low-income family, and the difficulties of wanting your child to be treated normally, while understanding the fragility of their physical and emotional well-being. We talked for hours and in a way it was a cathartic experience both for her and for me. The conversations I have had both on and off camera bring the film closer to understanding the uniqueness of managing life with a rare disease. 

Once you complete the editing the documentary, what are your plans for making it available for others to view ? Will the documentary be shown at any upcoming Film Festivals ?

With funds raised through our Indiegogo campaign, we hope to complete the editing and post-production by early spring 2015. After that we will work diligently on outreach and distribution, applying to a variety of well-established film festivals with the hopes of gaining a wider audience and working with distributors. Finally, we will make the film available online as well as on DVD for all to see!

Thank you Michaela for your time and we wish you the best with sharing your documentary with others.

Please Note: Photo courtesy of Michaela O’Brien.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.


Newborn Screening: MPS I Recommended To Be Added


The Health Resources and Services Administration (HRSA) is an agency of the United States Department of Health and Human Services (HHS), located in Rockville, Maryland. HRSA is the main U.S. federal agency with the responsibility of improving access to health care services for Americans who are uninsured, isolated, or medically vulnerable.

HRSA has Federal Advisory Committees, with the purpose of making recommendations or advising on issues relating to programs, responsibilities, and activities of HHS. One of these Federal Advisory Committees is the Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC), chartered in April 2013. DACHDNC’s mission is to:

“ … reduce morbidity and mortality in newborns and children who have, or are at risk for, heritable disorders. The Committee recommends that every newborn screening program include a Recommended Uniform Screening Panel (RUSP) that screens for 31 core disorders and 26 secondary disorders; the disorders’ selection was based on the Newborn Screening: Towards a Uniform Screening Panel and System.”  

DACHDNC met this month, February 12 – 13, and voted in favor of recommending to the Secretary of HHS, that MPS-1 be added to the RUSP.

MPS I is caused by a deficiency of an enzyme, alpha-L-Iduronidase. MPS I is also referred to as Hurler, Hurler-Scheie, and Scheie Syndromes. MPS I occurs in approximately 1/100,000 newborns globally.

Currently in the US, Aldurazyme (Laronidase) is approved by the FDA for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I), and for patients with the Scheie form who have moderate to severe symptoms. Aldurazyme is an Enzyme Replacement Therapy (ERT) injection for MPS I. In the European Union (EU), Aldurazyme is approved for MPS I to treat the non-neurological symptoms of the disease.

Please Note: “Blowing Bubbles at Sunset” by JaneArt (Own work) [CC-BY-SA-3.0 or GFDL] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

Biogen Idec And Academic Collaboration: Discovery Of New ALS Gene

Human Chromosomes

On February 19th, using advanced DNA sequencing methods, Biogen Idec announces that an international consortium, that includes clinicians and scientists from the company, Columbia University Medical Center (CUMC), and HudsonAlpha Institute for Biotechnology (Huntsville, Alabama) identify a new gene associated with sporadic Amyotrophic Lateral Sclerosis (ALS). The newly associated gene is called TBK1 (TANK-Binding Kinase1). It is also found that a gene, called OPTN, previously thought to play a minor role in ALS, may actually be a major player in the disease.

The study is published in the online edition of the journal Science with the following Abstract:

Amyotrophic lateral sclerosis (ALS) is a devastating neurological disease with no effective treatment. Here we report the results of a moderate-scale sequencing study aimed at identifying new genes contributing to predisposition for ALS. We performed whole exome sequencing of 2,874 ALS patients and compared them to 6,405 controls. Several known ALS genes were found to be associated, and the non-canonical IκB kinase family TANK-Binding Kinase 1 (TBK1) was identified as an ALS gene. TBK1 is known to bind to and phosphorylate a number of proteins involved in innate immunity and autophagy, including optineurin (OPTN) and p62 (SQSTM1/sequestosome), both of which have also been implicated in ALS. These observations reveal a key role of the autophagic pathway in ALS and suggest specific targets for therapeutic intervention.

The discovery of the new gene TBK1 for ALS, is important as it aids in identification of key biological pathways relevant to ALS that now can become the focus of targeted drug development. Several compounds that affect TBK1 signaling have already been developed for use in cancer, where the gene is thought to play a role in tumor cell survival.

Please Note: “Human Chromosomes” Courtesy of Jane Ades, NHGRI.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

FDA Breakthrough Therapy Designation: Celldex Therapeutics And Glioblastoma Vaccine


Celldex Therapeutics, a New Jersey-headquartered biotechnology company, announces February 23rd that the FDA grants the Breakthrough Therapy Designation (BTD) to the company’s investigational immunotherapy Rindopepimut (Rintega) vaccine, for adult patients with EGFRvIII-positive Glioblastoma (GBM). Only 3 drugs have been approved in more than 20 years for GBM. Rindopepimut receives Orphan Drug Designation (ODD) in Europe in 2011.

The FDA BTD is based on data from the Phase II ReACT clinical trial in relapsed EGFRvIII-Positive GBM, Phase II ACT III clinical trial in newly diagnosed GBM, and additional Phase II clinical trials. An international Phase III study, ACT IV, in newly diagnosed GBM completes enrollment in December 2014 with 745 participants.

Rindopepimut is an experimental cancer drug that is administered via intradermal injection. It acts to promote anti-cancer effects in patients who have tumors that express the EGFRvIII protein. Per the company’s Press Release:

Expression of EGFRvIII correlates with increased tumorigenicity in mouse models and poor long term survival in clinical studies of patients with glioblastoma (GBM). In addition, EGFRvIII-positive cells are believed to stimulate proliferation of non-EGFRvIII cells through IL-6 cell-to-cell signaling and to release microvesicles containing EGFRvIII, which can merge with neighboring cells, transferring tumor-promoting activity. EGFRvIII expression may also be associated with tumor stem cells that have been identified in GBM. These stem cells contribute to resistance to cytotoxic therapy and tumor recurrence. EGFRvIII is expressed in tumors in about 30% of patients with GBM. It has not been detected at a significant level in normal tissues; therefore, targeting of this tumor-specific molecule is not likely to impact healthy tissues.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “White Pocket Reflection” by John Fowler (Flickr: White Pocket Reflection) [CC BY 2.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.


Get every new post delivered to your Inbox.

Join 2,407 other followers

%d bloggers like this: