02012021

Mundipharma and Cidara’s rezafungin gets Orphan Drug Designation in EU
by Drug Discovery NewsRare Disease NewsRegulatory News

The European Commission (EC) has adopted the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) recommendation to grant Orphan Drug Designation (ODD) to Mundipharma and Cidara Therapeutics’ rezafungin for treating invasive candidiasis (IC).

IC is a severe, life-threatening Candida infection of the bloodstream and / or deep / visceral tissues.

A novel, once weekly echinocandin, rezafungin is currently in Phase III trials for treating severe fungal infections.

Mundipharma holds exclusive rights to develop and market the drug in all markets except the US and Japan, where Cidara retains the rights.

Mundipharma chief scientific officer Brian Sheehan said: “Orphan drug designation is an important milestone in the development of rezafungin, which is currently in Phase III clinical trials.

“Fungal infections still pose a major threat to the lives of hospitalised or immunocompromised patients. We are proud that patients affected with invasive candidiasis may have an additional treatment option to treat this potentially life-threatening condition.”

The latest development comes after rezafungin was granted the US Food and Drug Administration’s (FDA) Qualified Infectious Disease Product (QIDP) with Fast Track status and ODD for use in treating IC, including candidaemia.

Cidara Therapeutics president and CEO Jeffrey Stein said: “We are pleased by the decision of the EMA to grant orphan drug designation to rezafungin, further supporting its potential as the first new antifungal for the treatment of serious invasive Candida infections in nearly 15 years.”

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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02282020

Rare Disease Highlight: Persistent Corneal Epithelial Defects
by Rare Disease Highlight

Persistent Corneal Epithelial Defects (PCEDs) is a term used to describe individuals who have had an ocular wound to certain cells in the cornea (epithelial cells) that have not healed after 14 days 1. The estimated amount of individuals affected in the U.S. with a PCED is 73,434 – 99,465 persons, making it’s patient population rare 2. PCED is a condition that can develop from many different precursor complications including, but not limited to: persistent dry eye syndrome, limbal stem cell deficiencies, neurotrophic conditions, chemical burns, inflammation, herpes simplex disorders of the ocular region, diabetes, and others 1,3,4. Symptoms of PCEDs typically include ocular discomfort and vision difficulties 1. PCEDs can lead to the development of ulcers, scarring, and loss of vision. Risk factors that may increase the likelihood of developing PCEDs are older age, male gender, bacterial or viral infection, rheumatic diseases, or treatment with anticancer drugs 4. Some of the current treatments available include ocular lubrication, punctual plugs, bandage contact lens, and corneal transplants, while other medications are specific to the underlying cause of the PCED 1. Current treatments can be unsuccessful or cause negative side effects pointing to the need for newer more effective treatments to be developed to prevent the risk of further ocular complications and vision loss 3,5.

References

  1. Gupta S, Gupta P, Sayegh R. Healing a Persistent Corneal Epithelial Defect. In. EyeNet Magazine. American Academy of Ophthalmology 2014:33-35.
  2. Wirostko B, Rafii M, Sullivan DA, Morelli J, Ding J. Novel Therapy to Treat Corneal Epithelial Defects: A Hypothesis with Growth Hormone. Ocul Surf. 2015;13(3):204-212.e201.
  3. Napoli PE, Braghiroli M, Iovino C, Demarinis G, Fossarello M. A study of refractory cases of persistent epithelial defects associated with dry eye syndrome and recurrent corneal erosions successfully treated with cyclosporine A 0.05% eye drops. Drug Des Devel Ther. 2019;13:2001-2008.
  4. Vaidyanathan U, Hopping GC, Liu HY, et al. Persistent Corneal Epithelial Defects: A Review Article. Med Hypothesis Discov Innov Ophthalmol. 2019;8(3):163-176.
  5. Katzman LR, Jeng BH. Management strategies for persistent epithelial defects of the cornea. Saudi J Ophthalmol. 2014;28(3):168-172.

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

02212020

Rare Disease Highlight: Creutzfeldt-Jakob Disease
by Rare Disease Highlight

Creutzfeldt-Jakob disease (CJD) is a rapidly progressive, transmissible, and invariably fatal neurodegenerative brain disorder affecting both humans and animals. CJD belongs to a family of prion diseases or transmissible spongiform encephalopathies (TSE) and was first described in 1920 by Hans Creutzfeldt, later in 1923 by Alfons Jakob 1,2. The word “prion”, derived from proteinaceous infectious particle, was coined by Stanley B. Prusiner in 1982 after the discovery of the disease-associate protein 3. CJD is a very rare neurodegenerative disease with only 1,224 confirmed cases since 1979 in the US 4. Within a few months of infection, patients suffer from severe morbidities, including personality changes, cognitive impairment, uncontrollable muscle shaking, and lose the ability to move and speak, resulting in the death of the patient within one year of onset of illness 1,4. There are no pharmacological or surgical options for treatment. Instead, patients with CJD  are usually managed in an intensive care unit to allow symptomatic and supportive care 1. This leaves a significant unmet clinical need within these patients.

References

  1. Sitammagari, K. K. & Masood, W. Creutzfeldt Jakob Disease. StatPearls (StatPearls Publishing, 2018).
  2. Iwasaki, Y. Creutzfeldt-Jakob disease. Neuropathology 37, 174–188 (2017).
  3. Mackenzie, G. & Will, R. Creutzfeldt-Jakob disease: recent developments. F1000Research 6, 2053 (2017).
  4. CDC. Creutzfeldt-Jakob Disease. (2019). Available at: https://www.cdc.gov/prions/cjd/about.html. (Accessed: 20 February 2019)

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

02142020

Rare Disease Highlight: Narcolepsy
by Rare Disease Highlight

Narcolepsy is a rare disease that affects 1 in every 2,000 people worldwide 1. Narcolepsy is characterized by symptoms of excessive daytime sleepiness, hallucinations upon falling asleep and waking up, insomnia, sleep paralysis, and falling quickly into the rapid eye movement (REM) stage upon falling asleep 1,2. Narcolepsy is divided into two subtypes: Narcolepsy type 1 (N1) and type 2 (N2) 1,3. N1 is well understood and is thought to be autoimmune in nature 1,3,4. In addition to classic symptoms of narcolepsy, those with N1 almost always experience cataplexy – a phenomenon where a patient loses muscle control upon experiencing strong emotion. Those with N2 do not typically present with cataplexy, however, these patients can potentially experience cataplexy later in life, and their diagnosis will be changed accordingly 1,3.

Narcolepsy is a lifelong disorder presenting anywhere from childhood to young adulthood, however, an official diagnosis, which is confirmed by a sleep study completed in a sleep lab, may be delayed until adulthood 1,3,5. Misdiagnosis and comorbidities, as well as a lack of effective symptom recognition and awareness, may all lead to the average amount of time between symptom presentation and diagnosis being between 8-22 years 4,6. Treatment for narcolepsy is typically a combination of stimulants, wake-promoting medications, and sleep agents to help with insomnia and fragmented sleep patterns 1. Antidepressants and some sleep medications may also be used to treat cataplexy 1,2. Newer medications are being developed that promote wakefulness through more specific pathways, however, there is still no cure developed 1,2,3. There is also the question of reliability in sleep study results and subsequent diagnosis 5. These medication and diagnostic challenges point to a need for further research and development to create more effective medications and diagnostic tools for those affected by narcolepsy.

References

  1. Bhattarai J, Sumerall S. Current and Future Treatment Options for Narcolepsy: A Review. Sleep Sci. 2017;10(1):19-27.
  2. Burgess CR, Scammell TE. Narcolepsy: neural mechanisms of sleepiness and cataplexy. J Neurosci. 2012;32(36):12305-12311.
  3. Barateau L, Dauvilliers Y. Recent advances in treatment for narcolepsy. Therapeutic Advances in Neurological Disorders. 2019;12:1756286419875622.
  4. Golden EC, Lipford MC. Narcolepsy: Diagnosis and management. Cleveland Clinic Journal of Medicine. 2018;85(12):959.
  5. Lopez R, Doukkali A, Barateau L, et al. Test–Retest Reliability of the Multiple Sleep Latency Test in Central Disorders of Hypersomnolence. Sleep. 2017;40(12).
  6. Morse AM. Narcolepsy in Children and Adults: A Guide to Improved Recognition, Diagnosis and Management. Med Sci (Basel). 2019;7(12).

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

02072020

Rare Disease Highlight: Multiple Myeloma
by Rare Disease Highlight

Multiple Myeloma (MM) is a rare cancer that is characterized by the uncontrolled growth of white blood cells1-4. This uncontrolled growth is caused by changes and mutations to the DNA of the cells5,6. There are different subtypes of MM which are characterized by the number and type of mutations observed, along with prognosis associated with each subtype3,5,7,8. The underlying cause of MM is unknown, however, some individuals are at a higher risk for developing MM, including those who are of older age, male, African American, and those with a family history of MM2,4,9. MM is the second most common blood cancer and makes up about 1% of all cancer diagnoses2,4.  An early, inactive form of MM, called Smoldering Multiple Myeloma (SMM), usually occurs in patients before they are diagnosed with MM4,7,10. The difference between SMM and MM is that those with SMM won’t have visible symptoms, while MM patients will present with anemia, an increase in serum calcium, bone lesions, compromised immune function, kidney issues, and other complications4,10,11. The chances of SMM developing into MM is 10-20% each year7. Current treatments include stem cell transplant, chemotherapies, surgery, radiation, and novel therapies such as immunomodulatory agents and proteasome inhibitors12,13. Around 52.25% of patients diagnosed with MM survive 5 years past diagnosis14. Recent improvements in treatments have brought the survival rate of MM up, however,  there is still no cure, and current treatments are not always successful or have associated complications indicating that there is still a large need for investigations into newer and more effective treatments3,4,6.

References

  1. Kyrtsonis M-C, Koulieris E, Bartzis V, et al. Monoclonal Immunoglobulin. In: Multiple Myeloma – A Quick Reflection on the Fast Progress.2013.
  2. Halvarsson BM, Wihlborg AK, Ali M, et al. Direct evidence for a polygenic etiology in familial multiple myeloma. Blood Adv. 2017;1(10):619-623.
  3. Van Wier S, Braggio E, Baker A, et al. Hypodiploid multiple myeloma is characterized by more aggressive molecular markers than non-hyperdiploid multiple myeloma. Haematologica. 2013;98(10):1586-1592.
  4. Smith L, McCourt O, Henrich M, et al. Multiple myeloma and physical activity: a scoping review. BMJ Open. 2015;5(11):e009576.
  5. Fonseca R, Bergsagel PL, Drach J, et al. International Myeloma Working Group molecular classification of multiple myeloma: spotlight review. Leukemia. 2009;23(12):2210-2221.
  6. Chretien ML, Corre J, Lauwers-Cances V, et al. Understanding the role of hyperdiploidy in myeloma prognosis: which trisomies really matter? Blood. 2015;126(25):2713-2719.
  7. Prideaux SM, Conway O’Brien E, Chevassut TJ. The genetic architecture of multiple myeloma. Adv Hematol. 2014;2014:864058.
  8. Kazandjian D, Hill E, Hultcrantz M, et al. Molecular underpinnings of clinical disparity patterns in African American vs. Caucasian American multiple myeloma patients. Blood Cancer J. 2019;9(2):15.
  9. Alexander DD, Mink PJ, Adami H-O, et al. Multiple myeloma: A review of the epidemiologic literature. International Journal of Cancer. 2007;120(S12):40-61.
  10. Eslick R, Talaulikar D. Multiple myeloma: from diagnosis to treatment. Aust Fam Physician. 2013;42(10):684-688.
  11. Bladé J, Rosiñol L. Complications of Multiple Myeloma. Hematology/Oncology Clinics of North America. 2007;21(6):1231-1246.
  12. Mikhael J, Ismaila N, Cheung MC, et al. Treatment of Multiple Myeloma: ASCO and CCO Joint Clinical Practice Guideline. Journal of Clinical Oncology. 2019;37(14):1228-1263.
  13. Wallington-Beddoe CT, Pitson SM. Novel therapies for multiple myeloma. Aging (Albany NY). 2017;9(8):1857-1858.
  14. National Institute of Health. Cancer Stat Facts Web site. https://seer.cancer.gov/statfacts/html/mulmy.html. Published 2016. Accessed 02/05, 2020.

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

rarediseasehighlight

Rare Disease Highlight: Sanfilippo Syndrome
by Rare Disease Highlight

Named after Dr. Sylvester Sanfilippo in 1963, Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a lysosomal storage disease affecting the central nervous system of young children. This disease is caused by an autosomal recessive mutation in one of four enzymes required to break down large chains of sugar molecules called glycosaminoglycans (GAGs). These molecules are located in the cells of the nervous system and help build connective tissues in the body1. As a result of the genetic mutation, GAGs remain stored in the cells of the nervous system, causing progressive developmental damage. Sanfilippo syndrome often goes undetected for years and most children with the disease are born with no visible signs of the disease2. Symptoms such as developmental disabilities, movement disorders, significant hyperactivity, and seizures begin to appear after age 1, and learning abilities begin to slow between ages 2 and 63. Sanfilippo syndrome is rare, with an incidence of 1 in 70,000 births worldwide4. Because there is no cure or currently approved treatment, disease management or palliative care are implemented to treat symptoms, and life expectancy is typically around 15 years5.

Image by PublicDomainPictures from Pixabay

References used
  1. MPS III. MPS Society. https://mpssociety.org/learn/diseases/mps-iii/. Accessed July 18, 2019.
  2. Fedele AO. Sanfilippo syndrome: causes, consequences, and treatments. Appl Clin Genet. 2015;8:269-281. doi:10.2147/TACG.S57672
  3. Reference GH. Mucopolysaccharidosis type III. Genetics Home Reference. https://ghr.nlm.nih.gov/condition/mucopolysaccharidosis-type-iii. Accessed July 18, 2019. .
  4. Zelei T, Csetneki K, Vokó Z, Siffel C. Epidemiology of Sanfilippo syndrome: results of a systematic literature review. Orphanet J Rare Dis. 2018;13. doi:10.1186/s13023-018-0796-4
  5. Sanfilippo Children’s Foundation – What is Sanfilippo? https://www.sanfilippo.org.au/page/26/what-is-sanfilippo. Accessed July 18, 2019.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Rare Disease Highlight: Microvillus Inclusion Disease
by Rare Disease Highlight

Microvillus inclusion disease (MVID) is a congenital intestinal disorder characterized by chronic, severe, and watery diarrhea due to insufficient absorption of nutrients during digestion. Two forms of MVID have been identified: early-onset MVID, which develops within hours or days of birth, and late-onset MVID, which occurs in the first months of life1. Affected infants experience malnutrition, dehydration, developmental delays, and severe metabolic acidosis. The cause of MVID is unknown, however, the disease can be attributed to mutations in the MYO5B gene which result in abnormal microvilli, which reduce the intestine’s ability to absorb necessary nutrients and fluids. While the exact prevalence of MVID is unknown, the disease is extremely rare with less than 200 cases reported in Europe since it was first described in 1978 by GP Davidson3.  No medical treatment has been successful in overcoming MVID-associated intestinal failure. Affected infants remain dependent on total parenteral nutrition or intravenous replacement of fluid and electrolytes life-long4. Due to the severity of the disease, the prognosis for MVID is poor as a result of severe dehydration, metabolic imbalance, and sepsis5.

References used
  1. Microvillus inclusion disease. Genetics Home Reference. https://ghr.nlm.nih.gov/condition/microvillus-inclusion-disease. Accessed July 12, 2019.
  2. RESERVED IU–AR. Orphanet: Microvillus inclusion disease. https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Expert=2290&lng=EN. Accessed July 12, 2019.
  3. Towards understanding microvillus inclusion disease. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4733813/. Accessed July 12, 2019.
  4. Bunn SK, Beath SV, McKeirnan PJ, et al. Treatment of Microvillus Inclusion Disease by Intestinal Transplantation. Journal of Pediatric Gastroenterology and Nutrition. 2000;31(2):176.
  5. Ruemmele FM, Schmitz J, Goulet O. Microvillous inclusion disease (microvillous atrophy). Orphanet J Rare Dis. 2006;1:22. doi:10.1186/1750-1172-1-22

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

RWE Submissions: FDA Drafts Guidance
by Regulatory News

With an eye toward better tracking the use of real-world data (RWD) to generate real-world evidence (RWE), the US Food and Drug Administration (FDA) on Wednesday drafted guidance to help sponsors provide information to the agency on their use of RWD and RWE in a uniform format.

The uniform format is meant to help the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) track certain types of submissions using RWE under an investigational new drug application (IND), new drug application (NDA) or biologics license application (BLA).

Relevant submissions may include RWE used to support study objectives, such as: “IND submissions for randomized clinical trials that use RWD to capture clinical outcomes or safety data, including pragmatic and large simple trials; New protocols for single arm trials that use RWE as an external control; Observational studies that generate RWE intended to help to support an efficacy supplement; Clinical trials or observational studies using RWE to fulfill a postmarketing requirement to further evaluate safety or effectiveness and support a regulatory decision.”

But FDA also said in the 5-page draft that it does not intend to track RWE submissions that are not tied to a specific product or are not being used to support a regulatory decision regarding safety and/or effectiveness.

Such submissions that do not have to be identified as containing RWE include: “Natural history studies for development of a clinical outcome assessment or biomarker; Feasibility studies using RWE; Studies using RWD to perform exploratory analyses and generate hypotheses.”

In the cover letter accompanying a submission, FDA explains how the sponsor or applicant should identify the submission as containing RWE by including the following information: Purpose of using RWE as part of a regulatory submission (e.g. as part of a new product approval, to provide evidence to support a labeling change or as part of a postmarket commitment); study design using RWE (e.g. randomized or single-arm trial); RWD source(s) used to generate RWE.

Such RWD source(s) can include: “Data derived from EHRs; Medical claims and/or billing data; Product and/or disease registry data; Other data sources that can inform on health status (e.g., data collected from mobile technologies, patient-generated data).”

FDA explained that it will use this information for internal tracking purposes only.

The draft guidance also includes an appendix with a sample presentation of the cover letter for submissions including RWE.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Rare Disease Highlight: Idiopathic Pulmonary Fibrosis
by Rare Disease Highlight

Idiopathic pulmonary fibrosis (IPF) is the most commonly observed form of idiopathic interstitial pneumonia, an aging-associated, progressive, chronic, and irreversible lung disease with very limited therapeutic options 1,2. IPF is characterized by progressive scarring of the lungs causing respiratory failure and death. The exact cause of IPF is unclear, although there are some environmental and genetic risk factors to the disease including middle-to-older age, smoking, and chronic inflammation 1,3. Symptoms of IPF include shortness of breath after exertion combined with dry cough. As the disease progresses, discoloration of the skin and signs of right ventricular failure may occur together with respiratory failure, eventually leading to death 4.  Currently there are no approved therapies for the treatment of IPF, however supportive therapies are available to manage symptoms. IPF is rare, with an estimated incidence rate of 2.8-9.3 per 100,000 per year in North America and a low survival of only 20% for five years 5.

References used
  1. Pardo, A. & Selman, M. Lung fibroblasts, aging, and idiopathic pulmonary fibrosis. Ann. Am. Thorac. Soc. 13, S417–S421 (2016).
  2. Zieliński, M., Sitek, P. & Ziora, D. Idiopathic pulmonary fibrosis coexisting with lung cancer — a review. Adv. Respir. Med. (2015). doi:10.5603/ARM.a2018.0052
  3. Kaunisto, J., Salomaa, E., Hodgson, U., Kaarteenaho, R. & Myllärniemi, M. Idiopathic pulmonary fibrosis – a systematic review on methodology for the collection of epidemiological data. 1–11 (2013). doi:10.1186/1471-2466-13-53
  4. Sgalla, G. I., Biffi, A. L. & Richeldi, L. U. C. A. Idiopathic pulmonary fibrosis : Diagnosis , epidemiology and. 427–437 (2016). doi:10.1111/resp.12683
  5. Barratt, S. L., Creamer, A., Hayton, C. & Chaudhuri, N. Idiopathic Pulmonary Fibrosis ( IPF ): An Overview. 1–21 (2018). doi:10.3390/jcm7080201

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Rare Disease Highlight: Acute Myeloid Leukemia
by Rare Disease Highlight

Acute Myeloid Leukemia (AML) is a blood cancer in which blast cells (immature myeloid precursor cells) multiply uncontrollably within the bone marrow and blood.1 The accumulation of blast cells disrupts normal blood cell production in the bone marrow as well as blood distribution to the tissues. This disruption leads to symptoms such as chronic fatigue, anemia, a compromised immune system, and high risk of hemorrhage.4,5 In addition, the current treatments for AML can cause many additional side effects due to drug toxicity.6-8 As these combined effects are severe, AML patients have low overall survival despite treatment and can still relapse after recovery is observed.2,3 According to the Surveillance, Epidemiology, and End Results (SEER) program, there were 19,520 new cases of AML in the US in 2018, with a reported 5-year survival rate of 27.4%.9  Risk factors associated with AML include age, history of blood disease, and/or genetic disorders.2,3 Thus, an unmet need exists for AML therapies which are non-invasive, non-toxic, and do not result in relapse.

References used
  1. Spivak JL. Acute Myelogenous Leukemia (AML). Merck Manual 2017; http://www.merckmanuals.com/professional/hematology-and-oncology/leukemias/acute-myelogenous-leukemia-aml. Accessed November 20, 2017, 2017.
  2. Pui C-H, Carroll WL, Meshinchi S, Arceci RJ. Biology, Risk Stratification, and Therapy of Pediatric Acute Leukemias: An Update. J Clin Oncol. 2011;29(5):551-565.
  3. PDQ PTEB. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®). 2017.
  4. Cheng MJ, Hourigan CS, Smith TJ. Adult Acute Myeloid Leukemia Long-term Survivors. Journal of leukemia (Los Angeles, Calif). 2014;2(2):26855.
  5. Dohner H, Estey EH, Amadori S, et al. Diagnosis and management of acute myeloid leukemia in adults: recommendations from an international expert panel, on behalf of the European LeukemiaNet. Blood. 2010;115(3):453-474.
  6. Lipshultz SE, Sallan SE. Cardiovascular abnormalities in long-term survivors of childhood malignancy. J Clin Oncol. 1993;11(7):1199-1203.
  7. Volkova M, Russell R. Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment. Curr Cardiol Rev. 2011;7(4):214-220.
  8. Von Hoff DD, Layard MW, Basa P, et al. Risk factors for doxorubicin-induced congestive heart failure. Ann Intern Med. 1979;91(5):710-717.
  9. SEER. Cancer Stat Facts: Leukemia – Acute Myeloid Leukemia (AML). 2018; https://seer.cancer.gov/statfacts/html/amyl.html.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.