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Berlin: Orphan Drugs Access And Economics Masterclass





Mark your calendars: Berlin, Germany, will be the location of Fleming Europe’s pharmaceutical event, Orphan Drugs Access and Economics Masterclass, scheduled for June 9 – 10, 2015.

“Rare disease therapies would be highly unattractive under standard market conditions from an investor perspective. The small number of patients cannot financially justify expending resources into orphan therapy research and development, which is a long, expensive, and risky process. As the population of patients with a specific rare disease is very small, the investments must be recouped by increasing the price of the product. It has been acknowledged that, while regulatory incentives have stimulated research and development of orphan therapies on a global level, equitable and timely access to approved orphan medicinal products for rare disease patients remains an issue. Decisions on Pricing and Reimbursement are the exclusive competence of the Member States of the European Union …”


•   EU Regulation, Programs, & Mechanisms for Orphan Medicinal Products

•   Achieving Sustainable Access For Orphan Drugs

•   Methods for Economic Evaluation of Orphan Drugs

•   Alternative Ways to Raise Funds & Finance Orphan Drugs

•   Regulatory, Pricing & Reimbursement

•   Patients & Payers

•   Alternative Models: Drug Repurposing.

Speaker Panel

·   Carlos R. Camozzi, Chief Medical Officer, Orphazyme

·   Günter Harms, Market Access & Public Affairs Director, Shire

·   Camille Métais, Associate Director – Regulatory Affairs, Alexion Pharma International

·   Luis Cruz, General Manager Spain & Portugal, CSL Behring.

Please reference the Meeting Agenda for detail information, visit the website, or contact Martina Hrabinska at

Please Note: “Writing Hand” by gnuckx (Flickr: Roma Italy – Creative Commons by gnuckx) [Public domain or CC-BY-2.0] | Wikimedia Commons.

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

FDA Breakthrough Therapy Designation Approval Chart (Updated As Of 03/25/15)


This Blog Post updates both the FDA CBER and CDER BTD approval charts as of 03/25/15.

The Total FDA BTD Approval Chart is divided into 2 charts:

•   FDA CBER BTDs Receiving Approval

•   FDA CDER BTDs Receiving Approval.

FDA CBER BTDs Receiving Approval (as of 01/31/15)

The FDA CBER Breakthrough Therapy Designations receiving approval (as of 01/31/15), 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
Bexsero (Meningococcal Group B Vaccine) ***** 01.23.15 Novartis Meningococcal B Disease

***** Accelerated Approval

FDA CDER BTDs Receiving Approval (as of 03/25/15)

The FDA CDER Breakthrough Therapy Designations receiving approval (as of 03/25/15), 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
Blincyto (Blinatumomab) 12.03.14 Amgen Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (B-cell ALL)
Viekira Pak (Ombitasvir, Paritaprevir, Ritonavir) 12.19.14 AbbVie Chronic hepatitis C virus (HCV) genotype 1
Opdivo (Nivolumab) 12.22.14 Bristol-Myers Squibb Unresectable/ Metastatic Melanoma
Kalydeco (Ivacaftor) ** 12.29.14 Vertex Pharmaceuticals Cystic Fibrosis Ages 6 & older who have the R117H Mutation
Imbruvica (Ibrutinib) * 01.29.15 Pharmacyclics Waldenstrom’s Macroglobulinemia (WM)
Ibrance (Palbociclib) ***** 02.03.15 Pfizer Postmenopausal women with ER+/HER2- advanced breast cancer, as initial endocrine-based therapy for metastatic disease
Lucentis (Ranibizumab Injection) * 02.06.15 Genentech Diabetic Retinopathy (DR) with  Diabetic Macular Edema (DME)
Kalydeco (Ivacaftor) * 03.18.15 Vertex Pharmaceuticals Cystic Fibrosis Ages 2 to 5 who have 1 of 10 mutations in the CFTR gene
Eylea (Aflibercept Injection) * 03.25.15 Regeneron Pharmaceuticals Diabetic Retinopathy (DR) with  Diabetic Macular Edema (DME)

* Expanded the approved use

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

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

**** Pembrolizumab formerly known as Lambrolizumab

***** Accelerated Approval



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “IMG Carlingford Lough 0617” by Sarah777 at en.wikipedia [Public domain] | Wikimedia Commons.

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

Cincinnati Children’s Hospital Medical Center: NIH Award To Create National Rare Childhood Genetic Disorders Database


Cincinnati Children’s Hospital Medical Center (CCHMC) is awarded $2.2 million as part of the Newborn Screening Translational Research Network (NBSTRN), to create and maintain a national database of genetic information from children with rare genetic disorders. The national database is called the Longitudinal Pediatric Data Resource (LPDR). LPDR supports newborn screening researchers by offering permission-based access and data sharing for longitudinal collection of clinical and research information.

Among the list of diseases included in LPDR are:

•   Lysosomal Storage Disorders

•   Inborn Errors of Metabolism

•   Severe Combined Immunodeficiency Disorders.

Per Peter W. White, PhD, Director of the Division of Biomedical Informatics at CCHMC:

“A large number of serious diseases of newborns can potentially be identified through existing newborn screening programs. In a growing number of these disorders, early detection provides the opportunity for improving the lives of these children. The LPDR will fast-track research on these diseases by collecting much larger sets of patients, and following patient outcomes over time. This includes critical genomic data that can help identify mechanisms of disease, as well as novel intervention strategies.”


Cincinnati Children’s Hospital Medical Center March 24, 2015, Press Release.

Please Note:Dandelion Sisters” by US Nessie (Own work) [CC BY-SA 3.0 | via Wikimedia Commons.

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

2 FDA Breakthrough Therapy Approvals: Vertex Pharmaceuticals’ Kalydeco And Regeneron Pharmaceuticals’ Eylea




This Blog post reviews 2 FDA Breakthrough Therapy Designations (BTDs) that are approved this past week:

•   Vertex Pharmaceuticals’ Kalydeco (Ivacaftor)

•   Regeneron Pharmaceuticals’ Eylea (Aflibercept) Injection.

I – Vertex Pharmaceuticals’ Kalydeco

On March 18th, Vertex Pharmaceuticals receives FDA approval for Kalydeco for children ages 2 – 5 with Cystic Fibrosis (CF), who have 1 of the following 10 mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene:

1) G551D                           6) S1251N

2) G1244E                         7) S1255P

3) G1349D                         8) S549N

4) G178R                           9) S549R

5) G551S                          10) R117H.

Before last week’s approval, Kalydeco is approved in the US for patients ages 6+, with these 10 mutations. An additional 300 US children can now take Kalydeco. To meet the needs of this young age group, a “new weight-based oral granule formulation of Kalydeco (50 mg & 75 mg) that can be mixed in soft foods or liquids was created … ”. This allows children who may not be able to swallow a tablet to take Kalydeco.

II – Regeneron Pharmaceuticals’ Eylea

On March 25th, Regeneron Pharmaceuticals announces that the FDA approves Elyea injection for the treatment of Diabetic Retinopathy (DR) in patients with Diabetic Macular Edema (DME). Eylea is injected into the eye every month for 5 months, and then every 2 months.

In September 2014, Eylea receives the FDA BTD and Priority Review for this indication. Eylea is currently approved in the US, European Union (EU), and other countries, for the following indications:

•   Wet Age-Related Macular Degeneration (AMD)

•   Macular Edema following Central Retinal Vein Occlusion (CRVO)

•   Diabetic Macular Edema (DME)

•   Diabetic Retinopathy (DR) in patients with Diabetic Macular Edema (DME).

Eylea is a VEGF (Vascular Endothelial Growth Factor) inhibitor formulated as an injection for the eye. It blocks the growth of new blood vessels and also decreases the ability of fluid to pass through blood vessels in the eye.

Diabetic Retinopathy is a complication of diabetes that results in damaging the eye’s retina, which can lead to poor vision, vision loss, and other vision problems, such as:

“… DME, which refers to the swelling of the macula (the part of the retina responsible for central, fine vision) and progression to proliferative diabetic retinopathy, which often results in profound visual loss due to associated complications … DME is the most frequent cause of vision loss in patients with diabetes and eventually can lead to blindness”.

In February 2015, the FDA approved Genentech’s Lucentis (Ranibizumab), once-monthly injection, for the same indication – DR in patients with DME.



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 Breakthrough Therapy Designation: Statistics Chart Updated (as of 03/20/15)

FDA logo



The CBER BTD #s are current as of 02/28/15 and the CDER BTD #s are current as of 03/20/15.

The changes are as follows:

•   The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 285 to 291

•   The total # of FDA CDER + CBER BTDs Granted by the FDA remains the same at 82

•   The total # of FDA CDER + CBER BTDs Denied by the FDA increases from 154 to 155

•   The total # of FDA CDER + CBER BTDs Pending by the FDA increases from 49 to 54.

FDA CBER BTDs + CDER BTDs (as of 03/20/15)

Breakthrough Therapy  Designation (BTD) Category Total # of CBER Designations (07/09/12-02/28/15) Total # of CDER Designations (07/09/12-03/20/15) Total # of CBER +   CDER BTD Designations (07/09/12-03/20/15)
Total # of BTD Requests Received 47 244 291
Total # of BTDs Granted 11 71 82
Total # of BTDs   Denied 34 121 155
Total # of BTDs   Pending 2 52 54



•   28.2 % of the total # of BTD Requests Received results in the BTD being granted

•   53.3 % of the total # of BTD Requests Received results in the BTD being denied

•   18.5 % 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 © 2012-2015, Orphan Druganaut Blog. All rights reserved.

Rare Diseases: European Approval Of Stem Cell Therapy


In December 2014, the European Medicines Agency’s (EMA) Committee for Medicinal Products For Human Use (CHMP) adopts a positive opinion recommending a conditional marketing authorization for the medicinal product, Holoclar, a stem cell therapy for a rare disease. A conditional marketing authorization is given to a product when it fulfils an unmet medical need, when the benefit to public health of immediate availability outweighs the risk – additional clinical data is still required. In February 2015, the European Commission grants a marketing authorization valid throughout the European Union for Holoclar.

Holoclar is for the treatment of the rare eye condition (moderate to severe), Limbal Stem Cell Deficiency (LSCD), in adults due to ocular burns to the eye(s). It is the first medicine recommended for LSCD. Per the EMA website :

“Limbal stem cell deficiency is an eye condition in which the patient lacks cells called limbal stem cells, which are found at the edge of the cornea (the transparent layer in front of the eye) and which continuously renew and repair the cornea. The deficiency of limbal stem cells leads to clouding of the cornea and may result in impaired vision or blindness. Limbal stem cell deficiency is a long-term disease that is debilitating to patients because of the impaired vision and discomfort it causes.”

Holoclar, from Chiesi Farmaceutici S.p.A., is designated an orphan product by the EMA in November 2008. It is a living tissue equivalent, made from a patient’s undamaged area of the cornea and grown in the laboratory using cell culture. It is then transplanted into the affected eye(s) replacing the corneal cells, and results in the start of normal corneal cell growth and maintenance.

An interview, published this month in Nature, with Italian biologist Graziella Pellegrini, describes the 25-year struggle to develop Holoclar.

Please Note: Climbing to Kalindi Mountains of Himalayas, Uttarkashi, Uttarakhand, India” by Sharada Prasad CS from Berkeley, India (Almost There!) [CC BY 2.0] | Wikimedia Commons.

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

Orphan Drugs And Rare Diseases: Top 2014 IPOs On Western Stock Exchanges




This month, EvaluatePharma, publishes a Report titled, “Pharma & Biotech 2014 In Review”. The report reviews and analyzes the changes and trends in the pharma and biotech space for 2014. In particular, the report takes a look at the top Initial Public Offerings (IPOs). Some of the highlights of the Report on IPOs for 2014:

•   IPO filings doubled from 44 in 2013 to 87 in 2014

•   Of the 44 IPOs in 2013, 40 were US IPOs & 3 were European IPOs

•   Of the 87 IPOs in 2014, 73 were US IPOs & 13 were European IPOs

•   Total amount raised increased from $2.9 billion in 2013, to $6.3 billion in 2014.

The chart below identifies the top IPOs in the orphan drug and rare disease space for 2014. The data in the chart is from the EvaluatePharma Report (except for the last column which is from the Orphan Druganaut Blog).

Top 2014 Biotech IPOs On Western Stock Exchanges In Orphan Drug/Rare Disease Space

IPO Name Amount Raised (In Millions) Offering Price 2014 YE Change Since Float FDA Orphan Drug Designation/Rare Disease Indications In Development
Juno Therapeutics $304 $24 118% ODD  & Breakthrough Therapy Designation (BTD) in November 2014 For Acute Lymphoblastic Leukemia (ALL)
Kite Pharma $128 $17 239% ODD in March 2014 For Diffuse Large B-Cell Lymphoma; Cancer Immunotherapy Products
Versartis $126 $21 7% ODD in October 2013 For Growth Hormone Deficiency; Endocrine Disorders
ZS Pharma $123 $18 131% Hyperkalemia
Ultragenyx Pharmaceutical $121 $21 109% Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome), ODD in September 2011 for Hereditary Inclusion Body Myopathy (HIBM), X-Linked Hypophosphatemia (XLH); Genetic Diseases


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

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


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