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Erythropoietic Protoporphyria: An Unmet Medical Need for a Severe Photodermatosis

Erythropoietic protoporphyria (EPP) is a rare inherited disease of heme biosynthesis characterized by dermal photosensitivity which occurs secondary to a partial deficiency of ferrochelatase. In a phenotypically similar X-linked protoporphyria (XLP), there is overactivity of the heme biosynthesis enzyme, erythropoietic aminolevulinate synthase (ALAS2). Both EPP and XLP result in the accumulation of the photosensitizer protoporphyrin IX (PPIX) in erythrocytes, plasma, skin and liver. The accumulated PPIX is activated by sun exposure generating free radicals which result in tissue damage and severe pain.

EPP is a devastating lifelong disorder with very few evidence-based clinical management tools and no approved treatments in the United States. The number of patients affected by EPP in the US is unknown. In Europe, it  is estimated that about 1 in about 75,000 to 1 in 200,000 individuals are affected. Worldwide, EPP is the third most common porphyria, with an incidence of about 2 to 5 per 1,000,000 individuals. Diagnostic delay is not uncommon due to a lack of symptom awareness among pediatricians.

Clinically, EPP is characterized by both cutaneous and extracutaneous manifestation. Phototoxicity usually begins in infancy and manifests as intolerance to sunlight, including tingling, stinging, burning, itching and pain with an onset within minutes of exposure to sun or UV light. EPP can also be accompanied by lichenification, loss of lunulae, edema, erythema and petechiae. A phototoxic reaction typically lasts from hours to a few days, although reactions lasting 10 or more days have been reported, often accompanied by social isolation, excruciating pain, anxiety and sleep impairment.

Extracutaneous manifestations include PPIX-mediated bile duct obstruction, resulting in cholestatic liver injury in 20-30%, and eventually cholelithiasis, cholestatic hepatitis, fibrosis, cirrhosis and liver failure in 3-5% of patients. Progressive liver disease can require liver transplantation, although replacement of a diseased liver with the healthy liver can start the reaccumulation of PPIX. Thus, liver transplantation is not curative without a sequential liver and hematopoietic stem cell transplantation to correct the underlying enzymatic defect.

Absent effective treatments, photoprotection against UV-A and visible light, including sun avoidance are required to minimize cutaneous reactions and pain. Patients with EPP have to maintain a high level of vigilance about ambient and expected light conditions and undertake measures to minimize the risk of phototoxicity. Drastic lifestyle modification, including the use of protective clothing, skin foundation, shade, tinted windows and indoor living are essential to minimize the cutaneous sequelae of EPP. These symptoms, along with required behavioral modification markedly impact employment choices, socialization (sun avoidance), work or school attendance, productivity, activities of daily living and quality of life. Not surprisingly, one study reported that while most individuals with EPP were employed or attending school, almost half felt their choice of vocation had been influenced by their symptoms. Both lifestyle modification and sunlight avoidance can place a significant burden on families of individuals with EPP.

Although a number of treatments, including high dose β-Carotene, Vitamin C, N-Acetyl Cysteine and narrow band UVB therapy have been suggested in the literature, there is little evidence to support their use. The only evidence-based treatment for preventing phototoxicity in EPP is afamelanotide (Scenesse®), a controlled release subcutaneous implant (depot) from Clinuvel Pharmaceuticals, a Melbourne, Australia headquartered company. Afamelanotide, a structural analog of α-melanocyte stimulating hormone (α-MSH) was developed, patented and initially tested by Drs. Victor Hruby, Robert Dorr and (the late) Mac Hadley at the University of Arizona more than two decades ago. It was first envisaged developed as a sunless tanning agent. It is a melanocortin receptor agonist that binds predominantly to the melanocortin-1 receptor (MC1R), activating the synthesis of eumelanin which provides photoprotection through absorption of UV and visible light, scavenging of free radicals and increased superoxide dismutase availability.

Scenesse was approved by the EMA in December 2014 for prevention of phototoxicity in adult patients with EPP as a 16 mg subcutaneous implant administered every two months. The UK marketing authorization for Scenesse is under “exceptional circumstances” for ‘the prevention of phototoxicity in adult patients with EPP. Unfortunately, in 2017, the U.K. National Institute for Health and Care Excellence (NICE) concluded that Scenesse “is not recommended, within its marketing authorization, for preventing  phototoxicity in adults with erythropoietic protoporphyria” and this ruling was upheld in 2018 on appeal. This means that the National Health Service (NHS) will not reimburse for Scenesse in England. In reaching its decision, NICE made several observations about data on Scenesse submitted by Clinuvel, including treatment allocation unmasking (unblinding), a modest albeit statistically significant benefit in daylight exposure without pain (approximately 10 minutes per day) and the choice, validation, and relevance of quality of life instruments used. In June 2018, Clinuvel submitted and NDA for which FDA has assigned a PDUFA date of July 8, 2019 and granted Scenesse a Priority Review.

There is a need for improved early diagnosis to reduce sun exposure and provide appropriate follow up, including monitoring for hepatic complications. This can be done through improved education and point of care protoporphyrin testing. There is also a need for improved treatments with robust efficacy in EPP, preferably by the oral route for both cutaneous and extracutaneous manifestations. There is also a need for improved study designs that leverage our increased understanding of adaptive clinical trials, quality of life and health economic outcomes, evaluating the role of photoprovocation in clinical trials, and understanding the role of geographic and seasonal differences in natural sunlight duration and time spent outdoors as potential variables in timing, site selection and data analysis of EPP clinical trials.

Dr. Najib Babul, PharmD, MBA, is an entrepreneur and experienced pharmaceutical scientist, drug developer, inventor, author and consultant with over two decades of experience in bringing new and repurposed drugs to market. 

What Do Increased Overdose Deaths Tell Us About the Opioid Overdose Crisis?

In this post, I share a few thoughts about the most recent data on the opioid overdose crisisfrom Morbidity and Mortality Weekly Report (January 4, 2019) which show a 12% year over year increase in opioid overdose deaths (2016-2017).

The opioid overdose crisis continues to worsen and evolve due to the ongoing increase in fatalities involving synthetic opioids. Opioid Use Disorder (OUD) is a chronic relapsing illness characterized by repeated, compulsive opioid seeking or use despite harm. Opioid addiction generally refers to moderate to severe OUD, which the American Society of Addiction Medicine defines as “a primary, chronic disease of brain reward, motivation, memory, and related circuitry.”

More than 2.1 million Americans have an opioid use disorder (OUD), including 626,000with heroin related OUD. Overall life expectancy in the U.S. began to decline in 2015 due to the opioid overdose crisis, the first such decrease since the 1960s. According to a recent report from the Centers for Disease Control and Prevention (CDC) for the year 2017, 47,600 Americans died of opioid overdoses, a 12% increase over 2016. This means that on average, 130 Americans died each day from opioid overdoses in 2017, up from 116 daily opioid overdose fatalities in 2016. Synthetic opioids which include illicitly manufactured fentanyl and fentanyl analogs were involved in almost 60% of opioid overdose deaths, an increase of about 45% from the previous year. Overdose deaths from prescription opioids and heroin remained stable during 2016-2017.

A diagnosis of OUD confers a high risk for adverse medical consequences, including poor physical and mental health, increased health care utilization, a higher incidence of HIV and Hepatitis C, opioid overdose deaths and a 14-fold higher all-cause mortality than the general population. OUD is also associated with many adverse non-medical consequences, including higher rates of criminal behavior and incarceration, impaired social functioning, reduced labor force participation and lost productivity. The Council of Economic Advisers(CEA) estimated the economic cost of the opioid crisis at $504 billion in 2015. The CEA noted that previous economic cost estimates greatly undervalued the most important component of the loss - fatalities resulting from overdoses. The CEA estimate was derived from 2015 data, when opioid overdose mortality was 30% lower than the most recently available estimate (2017).

The increase in opioid overdose and deaths is not for lack of significant efforts on the part of operating divisions of the HHS, including the National Institute on Drug Abuse (NIDA), the Food and Drug Administration (FDA) and SAMHSA. These efforts have included the NIH HEAL Initiative, a public-private partnership in conjunction with the FDA to address the opioid crisis via more effective and safe ways to prevent and treat OUD and overdose; increased availability of the opioid overdose reversal agent naloxone; expanded access to medication-assisted treatments; and linking individuals with OUD to treatment and harm-reduction services. Without significant Congressional appropriations for the opioid overdose crisis, and intervention by various HHS operating divisions and other stakeholders at the Federal, State and local level, the devastation from the opioid overdose crisis would be far greater.    

Dr. Najib Babul, PharmD, MBA, is an entrepreneur, experienced pharmaceutical scientist, drug developer, inventor and consultant.

Pharmaceutical & Medical Device Regulatory Affairs Director

Collidion, Inc. and it’s subsidiaries,  Plex Pharmaceuticals & Avenlogics, Inc has an immediate full-time position open for Pharmaceutical & Medical Device Regulatory Affairs Director.

Position: Full-time

Location: Flexible

To apply, visit


Founded in 2015, Collidion, Inc. is a private healthcare company with two subsidiaries, Avenlogics, Inc. and Plex Pharmaceuticals. Collidion strives to improve the future of global healthcare through the acquisition, licensing, development, and commercialization of drugs and medical devices for various markets.  Collidion invests industry expertise and capital to develop these technologies into recognizable solutions for patients and medical professionals. Collidion values innovation, scientific rigor, collaboration and a passion for change.


Avenlogics, Inc., Collidion’s subsidiary, is focused on the development and commercialization of several anti-infective based products.  The Company’s pipeline of antimicrobial products addresses a growing need for alternative therapies to effectively treat infections caused by antimicrobial resistant pathogens. Collidion’s solutions include several antimicrobial platforms with potential applications in human and animal medicines, wound care, surface disinfection, and improved safety for the food supply chain. The company is located alongside with Collidion, Inc in Petaluma, CA.


Plex Pharmaceuticals is a scientifically-driven company with a vision to improve the lives of patients with complex diseases and conditions. The company is focused on the treatment of diseases generally caused by protein misfolding: ALS, Parkinson’s disease, and cataracts.  The company was founded in 2009.  Plex has a rich pipeline of drug discovery programs and a dedicated scientific team. Plex’s drug discovery lab and operations are located in San Diego, California.




  • Regulatory representation and leadership to cross-functional product development teams, development of regulatory strategies and support for clinical and non-clinical development programs and marketing applications. 
  • Responsible for project timelines and management of 510K, PMA, IND, NDA and global regulatory submissions. 
  • Lead regulatory activities including planning and reviewing of CMC, clinical and nonclinical sections of regulatory submissions. 
  • Plan, direct and prepare regulatory submissions (IND, NDA, IDE, 510(k), PMA) to ensure compliance with FDA and international regulations and guidelines. 
  • Develop and maintain SOPs with an emphasis on drug and device regulations. 
  • Perform research regarding regulatory strategic recommendations, and new and revised governmental regulations. 
  • Conduct successful negotiations and interactions with domestic and foreign regulatory agencies on assigned projects. 
  • Provide regulatory guidance with regard to preparation, review and approval of labeling and promotional materials. 
  • Prepare and assist with applicable regulatory audits. 
  • Statistical techniques. 





  • Minimum of B.S. in life sciences, engineering, or equivalent required. 
  • Advanced degree preferred. 





  • Minimum 5-7 years of experience in regulatory affairs for pharmaceutical and medical device biopharmaceutical companies. 
  • Veterinary product experience highly desirable. 
  • Experience with successful FDA and international agency negotiations and audits. 
  • Experience in developing and submitting successful 510(k), PMA, IND/NDA submissions with a thorough understanding of the drug and device development processes, FDA regulations and ICH guidelines. 
  • Excellent verbal and written communication skills. 





  • Detail oriented with well-developed organizational and analytical skills. 
  • Experience with submitting documents in CTD and eCTD format. 
  • Suited to working in a fast-paced, small company environment. 



Fully equipped 1000-1500 Sq. Ft. laboratory space available for sub-lease in Sorrento Mesa area

Plex Pharmaceuticals is interested in sub-leasing 1000-1500 Sq. Ft. of laboratory space on a month-to-month or long-term basis. The sub-lessee will have access to common laboratory equipment, chemical fume hood, tissue culture and other general lab instruments. Lab bench with 3-5 knee holes, dedicated office space, wi-fi, shared conference room, common kitchen and reception area. Interested parties please contact Sridhar Prasad at 858-733-0858 or email:

Attend Thomas Insel's talk: How Will Technology Change Healthcare? July 24, 2018 at 2:30pm

Presentation by Dr. Tom Insel on July 24th at the Illumina Theater, Alexandria at Torrey Pines. Appetizers and drinks sponsored by Neuropore Therapies.

About the event:

Dr. Insel will talk about leading edge technologies that may one day be of impact to the care of patients suffering from psychiatric conditions. He will discuss the advent of digital phenotyping, modern cognitive and linguistic assessments, and powerful computational approaches applied to complex data which now permit a deeper analysis of behavior than would have been possible even five years ago. Will this phenomic analysis in psychiatry serve the same role as molecular profiling in oncology? Will subgroups selected by modern phenomics be predictive of treatment response or clinical course? This presentation will argue that a detailed analysis of behavior and cognition combined with functional connectomics can become a pathway to precision medicine for psychiatry.

About Dr. Insel:

Thomas R. lnsel, M.D., a neuroscientist and psychiatrist, leads the Mental Health Team at Verily (formerly Google Life Sciences) in South San Francisco, CA. From 2002-2015, Dr. Insel served as Director of the National Institute of Mental Health (NIMH). In that role he also served as Chair of the Interagency Autism Coordinating Committee (IACC) as well as co-lead of the NIH BRAIN Initiative. Prior to serving as NIMH Director, Dr. lnsel was Professor of Psychiatry at Emory University where he was founding director of the Center for Behavioral Neuroscience and director of the Yerkes Regional Primate Center in Atlanta. Dr. Insel’s research has examined the neural basis of complex social behaviors, including maternal care and attachment. A member of the National Academy of Medicine, he has received numerous national and international awards and served in several leadership roles at NIH.

Networking after:

Don't miss the networking event outside the Illumina Theater right after the presentation. Drinks and appetizers are sponsored by Neuropore Therapies, Inc.

About Sponsor:

Neuropore Therapies, Inc. is a San Diego, California based biopharmaceutical company that is focused on the discovery and development of novel small molecule therapeutics for the treatment Parkinson’s disease and other neurodegenerative disorders. The approaches being taken by Neuropore Therapies target the core pathological processes underlying these disorders including the intracellular accumulation of neurotoxic misfolded proteins and the associated neuroinflammation. As such, the therapeutics being developed by Neuropore may provide the first effective means for slowing the unremitting progression of these devastating diseases.

Learn more and register here

Neuropore Therapies receives 2nd grant from The Michael J. Fox Foundation for Parkinson's research!

The Michael J. Fox Foundation for Parkinson's research awarded Neuropore Therapies a second grant on its TLR2 antagonist Program for Parkinson's disease.  READ MORE:

Michael Swit announces re-launching of his FDA regulatory law practice.

The Law Offices of Michael A. Swit, devoted to Solving FDA Legal Challenges for the Life of a Life Sciences Company

Please visit our website at

Happy Holidays!

Looking for Lab Clean Room 2,000-3,000 square foot

An international bio-tech company is looking urgently for lab space (plus office space of 2,000-3,000 square foot) to buy or lease. They would prefer a setting where the clean room space is already built, but will consider any location where the building is properly zoned and has the utilities to support tenant improvement.

As a member of SDEE I would highly appreciate any help from the community. Please reach out to me at if you have any information on potentially available lab space.

Fibromyalgia Therapeutics: Why has progress has been slow?

Fibromyalgia is a common medical condition characterized by chronic widespread muscle pain, fatigue, and sleep disturbance affecting two to three percent of the total population. It is five to ten times more common in women than men, increases with age and can also be worsened by many factors, including physical trauma and emotional stress. In this post, I share a few thoughts on why advances in the field have been slow and what developments may be coming soon.

Fibromyalgia involves central amplification of peripheral sensory signals, so that otherwise normal sensations are perceived as painful. Most patients afflicted with fibromyalgia also experience excessive tenderness, fatigue, sleep disturbances, problems concentrating, and functional impairment. A major obstacle to diagnosis is the absence of specific laboratory or imaging tests for fibromyalgia, although lab testing can be used to rule out other conditions associated with fatigue. Since the symptoms of fibromyalgia are shared with other conditions, patients should also be evaluated for mood disorders and sleep impairment.

In the decades prior to the introduction of the American College of Rheumatology’s (ACR) classification criteria in 1990, there was considerable skepticism about fibromyalgia as a legitimate medical condition. The 1990 ACR criteria ushered in an era of increased recognition of fibromyalgia and provided a standardized diagnostic approach. This was a boon for academic rheumatologists and even more so for researchers looking to test the efficacy of drugs. Despite widespread dissemination of the diagnostic criteria, skepticism abounded. For example, the very year ACR published the criteria for fibromyalgia, the British Journal of Rheumatology and the Canadian Family Physician journal published papers questioning the existence of fibromyalgia. However, the ACR criteria provided a major boost to research, resulting in the 2006 FDA approval of pregabalin (Lyrica®); the 2008 approval of duloxetine (Cymbalta®); and the 2009 approval of milnacipran (Savella®), which remain the only FDA-approved medicines for the treatment of fibromyalgia.

Although the ACR classification system was a major advance, it proved problematic in a number of ways. First, it required assessment of tenderness on pressure (tender) points in at least 11 of 18 specified sites and the presence of widespread pain for a diagnosis of fibromyalgia. Second, tender point assessment failed to address the symptoms that bothered patients. And finally, digital palpation of 18 tender points was hard for most non-rheumatologists to perform. It became apparent that primary care physicians had neither the time nor the training to perform extensive tender point assessment. Therefore, in 2010, the ACR simplified its criteria, eliminating tender point assessment, and changed the definition of fibromyalgia to an illness characterized by self-reported multiple painful regions and core symptoms such as such as fatigue, sleep disturbances and impaired cognition. In 2011, the ACR provided a self-report version for survey and clinical research purposes, thus spurring further testing of treatments for fibromyalgia. In December 2016, ACR released a further revision to the 2010/2011 criteria to reduce misclassification of regional pain disorders and improve diagnostic clarity. The 2016 modifications provide physician-based criteria for patient diagnosis and self-report criteria for research studies.

Although there are no definitive treatments for fibromyalgia, evidence indicates that lifestyle changes including relaxation, patient education, counseling, and exercise all improve symptoms and should therefore be an active part of any management program. In recent years, several promising drug treatments have failed in clinical trials, although companies such as Astellas, Daiichi Sankyo, Zynerba and SWITCHBIOTECH are pursuing new treatments for fibromyalgia. As awareness is raised and research studies continue, the contributing causes of fibromyalgia will be better understood and allow for the development of superior treatments. Najib Babul, PharmD, MBA

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