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Europe’s November 2014 Products Recommended For Orphan Drug Designation


The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting November 11– 13, 2014.

At this meeting, there are 27 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are forwarded to the European Commission (EC). The EC will then decide whether to grant an orphan designation for the medicines in question. Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC.

EMA COMP November 2014 ODDs Recommended

Product Name Sponsor Company Indication
Adeno-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA Lysogene Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Exisulind Cell2B Advanced Therapeutics Prevention of graft-versus-host disease
4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid Firc Institute of Molecular Oncology (IFOM) Familial cerebral cavernous malformations
Heat-killed Mycobacterium obuense (whole cell) Immodulon Therapeutics Ltd Pancreatic Cancer
Single-chain urokinase plasminogen activator Coté Orphan Consulting UK Limited Pleural empyema
((E)-1-(4′-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one) Centre National de la Recherche Scientifique (CNRS) WHIM syndrome
(1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine MIMETECH S.r.l. Neurotrophic keratitis
1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one AstraZeneca AB Multiple system atrophy
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one Aprea AB Ovarian Cancer
5,5’-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol Prof. Olivier Blin Glioma
5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine TMC Pharma Services Ltd Malignant mesothelioma
5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl) pyrimidine-4,6-diamine Palobiofarma S.L. Huntington’s disease
Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain Alan Boyd Consultants Ltd Glioma
Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist Novartis Europharm Limited acute lymphoblastic leukaemia
Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells IPD-Therapeutics BV acute myeloid leukaemia
Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis Biodan Yelah S.L. Epidermolysis bullosa (EB)
Amikacin sulfate PlumeStars s.r.l. Pseudomonas aeuriginosa lung infections in cystic fibrosis
Autologous collagen type II-specific regulatory T cells TxCell Non-infectious uveitis
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor Kite Pharma UK, Ltd Diffuse large B cell lymphoma
Benserazide hydrochloride Isabelle Ramirez Beta-thalassaemia intermedia and major
Bevacizumab Dr Sophie Dupuis-Girod Hereditary haemorrhagic telangiectasia
Chenodeoxycholic acid Sigma-Tau Pharma Ltd Inborn errors in primary bile acid synthesis
Edaravone Treeway B.V. Amyotrophic lateral sclerosis
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor Oncos Therapeutics Oy Malignant mesothelioma
Pegylated recombinant human hyaluronidase PH20 Pharm. Research Associates (UK) Limited Pancreatic cancer
Plerixafor Groupe d’étude des neutropénies WHIM syndrome
Riluzole Dr Laurent Vinay Traumatic spinal cord injury


Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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

Introducing 1st Rare Disease Advocacy World (RDAW) USA 2015 Meeting

RDAW launch 200x200

Patient advocacy groups are one of the most important powerful stakeholders that contribute to the success of orphan drug development and changes in policies for global regulatory agencies.

Just recently in September 2014, the European Medicines Agency (EMA) announces the launch of a pilot program to involve patients in the benefit-risk evaluation  of medicines at the regulatory agency’s Committee for Medicinal Products for Human Use (CHMP). CHMP is the EMA committee responsible for preparing the agency’s opinions on questions concerning human medicines – it conducts the initial benefit-risk analysis of medicines. In August 2014, Rona Ambrose, Canada’s Minister of Health, announces the launch of a pilot project “ … targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs …. The Pilot Project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect.” The FDA has started the Patient-Focused Drug Development (PFDD) initiative to obtain and better understand a patient’s perspective about their disease and to get patient feedback on treatments.

In recognition of the power, strength, passion, and compassion of patient advocacy organizations, there is a new portion of the World Orphan Drug Congress USA 2015, devoted to this trend. Rare Disease Advocacy World (RDAW) is a conference of its own which leverages the success of the world’s largest orphan drug event – The World Orphan Drug Congress USA – to grow the breadth and scope of the event even further and embrace and empower rare disease advocacy like never before. RDAW brings stakeholders together at this co-located conference. RDAW will be a 2-day event that will provide opportunities for patient advocacy groups to learn more about:

•   Achieving a scientific mindset

•   Understanding your disease

•   Characterizing your patients

•   Developing a strategic plan

•   Raising awareness and raising funds.

Several experts in the field will be hosting a series of case studies and workshops:

•   Pat Furlong, President & CEO, Parent’s Project Muscular Dystrophy (PPMD)

•   Nancy Goodman, Founder & Executive Director, Kids V Cancer

•   Eric Hoffman, Director, Research Center for Genetic Medicine, The George Washington University.

A Brochure is available for download.

Rare Disease Advocacy World (RDAW) Logo courtesy of Terrapinn. The Orphan Druganaut Blog is a Media Partner.

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

Ultra-Rare Diseases: BioBlast Pharma Orphan Drug Designations


BioBlast Pharma, a Tel Aviv, Israel-based clinical-stage biotech founded in 2012, is developing a platform of products for rare and ultra-rare genetic diseases.

I – Cabaletta For Oculopharyngeal Muscular Dystrophy (OPMD)

BioBlast Pharma’s most advanced product Cabaletta, a mutant protein stabilizing platform, is in a Phase II clinical trial (HOPEMD) for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD). Cabaletta is an IV formulation of Trehalose, which the company says enables the drug to reach muscles. Trehalose prevents aggregation of proteins in cells. BioBlast Pharma receives FDA Orphan Drug Designation (ODD) in October 2013 for Cabaletta (Trehalose) for the treatment of OPMD.

OPMD is a rare genetic condition and form of muscular dystrophy that begins usually after age 40. It effects the muscles of the eyelid first, resulting usually in droopy eyelids (ptosis), followed by problems with swallowing (dysphagia). Patients with OPMD can also have weakness in the muscles near the center of the body (proximal muscles). There is currently no medical therapy that can alleviate symptoms or slow the progression of OPMD.

OPMD is caused by a genetic defect in a gene that encodes a protein called PABPN1. The mutation can be diagnosed by a molecular blood test.

II – Cabaletta For Spinocerebellar Ataxia Type 3 (SCA3)

BioBlast Pharma announces on November 19th, that Trehalose receives FDA ODD for the treatment of Spinocerebellar Ataxia Type 3 (SCA3). This is the 2nd FDA ODD for Cabaletta. A Phase II clinical trial to test the efficacy of Cabaletta in SCA3 is currently being conducted and a SCA3 pivotal study is planned to start in 2015.

SCA3 is a genetic disease where patients have memory deficits, difficulty with swallowing and speech, and other muscular disorders. SCA3 begins in early adolescence and progresses over time. It is an incurable disease with no currently approved treatment. SCA3 is caused by an expansion of a gene called ATXN3. Currently there is no treatment that is effective.

BioBlast Pharma FDA ODDs For Cabaletta

# FDA ODD Date Indication
1 10.25.13 Occulopharyngeal Muscular Dystrophy (OPMD)
2 11.17.14 Spinal Cerebellar Ataxia type 3 (SCA3 or Machado Joseph disease)


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

FDA Breakthrough Therapy Designation: Pfizer’s Meningococcal B Vaccine Available In US



Pfizer announces November 18th that Trumenba (Meningococcal Group B Vaccine) is now available for healthcare providers, retail pharmacies, hospitals and college health centers to order in the US. It is the first and only FDA-approved vaccine for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroup B in people ages 10 – 25.

Just 3 weeks ago on October 29th, the FDA grants Accelerated Approval of Trumenba. Trumenba is reviewed and approved under the FDA’s Breakthrough Therapy Designation (BTD) and Priority Review programs. Pfizer receives the FDA BTD in March 2014. Part of the FDA Accelerated Approval process requires Pfizer to conduct further studies to verify Trumenba’s effectiveness against additional strains of N. meningitidis serogroup B.

Pfizer’s competitor for this indication in the US is Novartis’s Bexsero Vaccine that receives the FDA BTD in April 2014 Bexsero is not yet approved of in the US. In Europe, Australia, and Canada, Bexsero is already approved for Meningococcal disease caused by serogroup B.   

Please Note: “Vaccine” by John Keith (Photographer) [Public domain, NIH], via Wikimedia Commons | Wikimedia Commons.

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

2014 Immunotherapy Cancer Biotech Initial Public Offerings (IPOs)


In addition to Juno Therapeutics receiving FDA Orphan Drug Designation (ODD) for its lead product candidate JCAR015 for Acute Lymphoblastic Leukemia (ALL) last week, the company files a S-1 Form with the US Securities and Exchange Commission (SEC) Commission on November 17th. The S-1 proposes an Initial Public Offering (IPO) of its common stock raising up to $150 million. The company’s listing will be under the symbol “JUNO” on the NASDAQ Global Select Market. Morgan Stanley, J.P. Morgan and Goldman Sachs & Co are underwriting the IPO.

Per the S-1 Form, as of July 2014, JCAR015’s most recent data demonstrates, in an ongoing Phase I clinical trial, an amazing 91% complete remission rate in 22 evaluable adult patients with relapsed/refractory B cell ALL. Historical complete remission rates without JCAR015 in a similar population are less than 10%. JCAR015 Phase I trials are currently underway at Juno’s collaboration partner, Memorial Sloan-Kettering Cancer Center. The company plans on starting a Phase II trial in mid-2015 exploring JCAR015 in the same population that could support accelerated US regulatory approval.

By the end of 2015, Juno Therapeutics plans to start testing for at least 3 more drug candidates.

Other biotechnology IPOs in 2014 developing cancer immunotherapies:

•   Kite Pharma (partnering with the US National Cancer Institute); Receives FDA ODD in March 2014 For Diffuse Large B-Cell Lymphoma (DLBCL) & EMA COMP positive opinion recommendation for ODD on November 17th

•   Affimed Therapeutics

•   Immune Design

•   Argos Therapeutics

•   Bellicum Pharmaceuticals (files 11/18/14 registration statement for proposed IPO with the US SEC to raise up to $115 million).

Please Note: “Wall Street & Broadway” by Fletcher6 (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

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

Top 9 Fastest Growing Orphan Drugs For Past Year



FirstWord Pharma, a supplier of global news and intelligence to the pharmaceutical industry, publishes a list of the top 50 biggest growing drugs for the past 4 quarters (4th Quarter 2013 through the 3rd Quarter 2014). The top biggest growing drugs over the past 4 quarters or year delivers “an absolute increase in sales worth $31 billion.”

Some interesting observations are presented in the article:

•   26% of the combined growth generated by the top 50 drugs was made by Gilead Sciences’ Hepatitis C non-orphan drug, Sovaldi, (FDA Breakthrough Therapy Designation (BTD) receiving approval in December 2013)

•   Regeneron Pharmaceuticals’ FDA BTD ophthalmology non-orphan drug Eylea (Afibercept) is the 5th fastest growing drug.

Top 9 Fastest Growing Orphan Drugs For The Past 4 Quarters

The chart below identifies the top 9 orphan drugs from the list of the top 50.

FirstWorld Pharma Ranking Product Sponsor Company Combined Sales of Last 4 Quarters In$ Millions Growth (versus previous 4 Quarters)In $ Millions
3 Humira AbbVie $12219 $ 1899
6 Remicade J&J / Merck $  9343 `$  679
9 Revlimid Celgene $  4794 `$  648
10 Soliris Alexion Pharmaceuticals $  2076 $  646
22 Pomalyst Celgene $    599 $  414
26 Imbruvica J&J / Pharmacylics $    322 $  322
31 Yervoy Bristol-Myers Squibb $  1202 $  291
33 Afinitor Novartis $  1510 $  289
36 Sprycel Bristol-Myers Squibb $  1460 $  264

Source of Data: Company Reported Data (FirstWorld Pharma article)


Please Note: “3D Bar Graph Meeting” by “The Gold Guys Blog” [CC-BY-SA-2.0] via Wikimedia Commons.

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

FDA Breakthrough Therapy Designation CBER And CDER Approval Charts

FDA logo

Starting November 17th, 2014, the FDA BTD Approval Chart will be divided into 2 charts:

•   FDA CBER BTDs Receiving Approval

•   FDA CDER BTDs Receiving Approval.

FDA CBER BTDs Receiving Approval (as of 10/29/14)

The FDA CBER Breakthrough Therapy Designations receiving approval (as of 10/29/14), in ascending chronological “FDA Approval Date” order, are as follows:

Drug Name FDA Approval Date Sponsor Company Indication
Trumenba (Meningococcal Group B Vaccine) ***** 10.29.14 Pfizer Meningococcal B Disease

***** Accelerated Approval.

FDA CDER BTDs Receiving Approval (as of 10/15/14)

The FDA CDER Breakthrough Therapy Designations receiving approval (as of 10/15/14), in ascending chronological “FDA Approval Date” order, are as follows:

Drug Name FDA Approval Date Sponsor Company Indication
Gazyva   (Obinutuzumab) 11.01.13 Genentech Chronic Lymphocytic Leukemia (CLL)
Imbruvica   (Ibrutinib) 11.13.13 Pharmacyclics Mantle Cell Lymphoma (MCL)
Sovaldi   (Sofosbuvir) 12.06.13 Gilead   Sciences Hepatitis C
Kalydeco (Ivacaftor)** 02.21.14 Vertex Pharmaceuticals 8 additional mutations in CFTR gene for Cystic Fibrosis (CF)
Arzerra (Ofatumumab) *** 04.17.14 GlaxoSmithKline In combination with Chlorambucil for previously untreated Patients with CLL for whom fludarabine-based therapy is considered inappropriate
Zykadia (Ceritinib) 04.29.14 Novartis Metastatic ALK+ NSCLC
Zydelig (Idelalisib) 07.23.14 Gilead Sciences Chronic Lymphocytic Leukemia (CLL)
Imbruvica (Ibrutinib)* 07.28.14 Pharmacyclics Chronic Lymphocytic Leukemia (CLL) who carry a deletion in chromosome 17 (17p deletion)
Promacta (Eltrombopag)** 08.26.14 GlaxoSmithKline Severe Aplastic Anemia (SAA) with insufficient response to Immunosupporessive Therapy (IST)
Keytruda (Pembrolizumab) **** 09.04.14 Merck Unresectable/ Metastatic Melanoma, disease progression, & have BRAF V600 mutation, following treatment with Yervoy & a BRAF inhibitor
Harvoni (Ledipasvir /Sofosbuvir) 10.10.14 Gilead Sciences Genotype 1 Chronic HCV
Esbriet (Pirfenidone) 10.15.14 InterMune Idiopathic Pulmonary Fibrosis
Ofev (Nintedanib) 10.15.14 Boehringer Ingelheim Pharmaceuticals Idiopathic Pulmonary Fibrosis

* Expanded the approved use of Imbruvica (Ibrutinib) for CLL

**   Approval for a Supplemental New Drug Application (sNDA)

*** Approval for a Supplemental Biologic License Application (sBLA)

**** Pembrolizumab formerly known as Lambrolizumab

Please Note: FDA Official Logo from FDA website.

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


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