Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market?

Dystrophic epidermolysis bullosa (DEB) is a common skin disease with a chronic history and low healing rates. Despite current management therapies, the disease has the potential to become severe and devastating for many patients over time. Dystrophic epidermolysis bullosa is classified into six types: dominant dystrophic epidermolysis bullosa (DDEB), recessive dystrophic epidermolysis bullosa (RDEB), RDEB-generalized severe (GS), RDEB-generalized intermediate (GI), RDEB-other, and RDEB-unknown subtypes. The dominant dystrophic epidermolysis bullosa (DDEB) accounted for the greatest number of cases in the 7MM in 2021.

As per our analysis, the total diagnosed dystrophic epidermolysis bullosa prevalence in the 7MM countries was estimated to be close to 3.5K cases in 2021. Moreover, in 2021, the total dystrophic epidermolysis bullosa prevalence was highest in the US. Additionally, as per DelveInsight’s analysis, the highest cases of dystrophic epidermolysis bullosa were found in the 1-9 age group in 2021 in the United States, and it is estimated that the dystrophic epidermolysis bullosa prevalence will increase by 2032.

Changing Dynamics of Dystrophic Epidermolysis Bullosa Treatment Landscape

The current dystrophic epidermolysis bullosa treatment includes contemporary wound care and the reduction of external factors that cause blistering and hinder wound healing. A high degree of personal hygiene and intense moisturizing dystrophic epidermolysis bullosa treatment is required for proper skin care. Blisters that do not have subsequent damage or infection heal nicely with regular skin care and cleanliness. Aqueous disinfectants of today are very effective and comfortable to use. Adhesives and compressive dressings should be avoided since they promote the formation of new blisters. Silicon-based, mildly adherent wound care dystrophic epidermolysis bullosa treatments have shown to be quite beneficial, especially for harsh skin regions such as the elbows, shoulders, and trunk.

Nonadhesive semiocclusive dressings, such as soft silicone and foam dressings, are preferred for dystrophic epidermolysis bullosa treatment because they absorb exudate and reduce pain and blister development during dressing changes. Nonadhesive soft silicone or lipido-colloid contact layers, such as Mepitel (Mölnlycke Healthcare), Adaptic touch (Systagenix), Urgotul (Urgo Medical), or Silflex (Advancis Medical), are the most acceptable primary dressings to utilize for dry to gently oozing chronic epidermolysis bullosa wounds.

Moreover, hydrofiber dressings such as Aquacel (Convatec) or a soft silicone foam with super-absorbers such as Cutimed Siltec (BSNmedical) may be useful in dystrophic epidermolysis bullosa treatment in cases of more severely leaking wounds. PolyMem (Ferris) is a polymeric membrane bandage that, when colonized or infected, absorbs considerable volumes of exudate and produces a surfactant to wash the wounds. For infected epidermolysis bullosa wounds, silver-containing dressings such as Mepilex Ag (Mölnlycke Healthcare), PolyMem Silver (Ferris), and Urgotul SSD (Urgo Medical) have also been used for dystrophic epidermolysis bullosa treatment for short periods of time.

Furthermore, moisturizers containing an antibacterial ingredient, such as benzalkonium chloride or chlorhexidine dihydrochloride, which are both present in dermol products (Dermal Laboratories), can aid in bacterial colonization reduction. Maceration and secondary skin degradation can be reduced by using barrier preparations such as Cavilon (3M) or Proshield Plus cream (H&R Healthcare). Care must be exercised while administering emollients to newborns.

However, systemic dystrophic epidermolysis bullosa treatment is usually not indicated. Systemic antibiotics (tetracycline) or corticosteroids may be prescribed for brief periods of time. Pediatricians, dermatologists, gastroenterologists, hand surgeons, dentists, dietitians, physical therapists, psychiatrists, and other professionals must collaborate to handle secondary dystrophic epidermolysis bullosa symptoms in many organs in seriously afflicted individuals with extra-cutaneous involvement.

Moreover, several dystrophic epidermolysis bullosa companies such as Castle Creek Biosciences, Amryth Pharma, Krystal Biotech, Abeona Therapeutics, BridgeBio, Phoenix Tissue Repair, Lenus Therapeutics, and others are evaluating their lead assets for dystrophic epidermolysis bullosa treatment. 

Promising Therapeutics Options for Dystrophic Epidermolysis Bullosa Treatment

There are currently no authorized drugs for dystrophic epidermolysis bullosa treatment. The current dystrophic epidermolysis bullosa treatment just alleviates symptoms; it does not give a long-term cure. As a result, there is an urgent need for more effective and tailored solutions with improved long-term side effect profiles.

According to DelveInsight's expert analysts, the dystrophic epidermolysis bullosa market size is likely to rise positively throughout the forecast period (2022–2032), owing to the growing dystrophic epidermolysis bullosa prevalence and the anticipated launch of innovative dystrophic epidermolysis bullosa therapies. As per our analysis, the dystrophic epidermolysis bullosa market size was USD 500 million in the 7MM in 2021.

Moreover, the market has a promising outlook with the emerging dystrophic epidermolysis bullosa therapies. Some of the drugs in the dystrophic epidermolysis bullosa pipeline include FCX-007 (Castle Creek Biosciences), Oleogel (Amryt Pharma), B-Vec (Krystal Biotech), EB-101 (Abeona Therapeutics), PTR-01 (BridgeBio), RGN-137 (Lenus Therapeutics), and others.

FCX-007 (Castle Creek Biosciences) is an investigational dystrophic epidermolysis bullosa gene therapy being tested in individuals with DEB to treat a lack of functional type VII collagen protein (COL7). Currently, it is being studied in phase III (DeFi-RDEB), a randomized, controlled, open-label, multicenter dystrophic epidermolysis bullosa clinical trial to examine if administering FCX-007 in addition to standard of dystrophic epidermolysis bullosa treatment promotes wound healing in children, adolescents, and adults with RDEB.

FCX-007 has been designated as an orphan drug by the US FDA and the European Medicines Agency (EMA). Furthermore, the FDA has given FCX-007 Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations for recessive dystrophic epidermolysis bullosa treatment.

Oleogel (Amryt Pharma) is a herbal medicine that contains birch triterpenes derived from birch bark. The medication is a keratinocyte modulator; transient receptor potential channel stimulants are used to accelerate wound healing in adults and children with DEB.

The FDA issued a Complete Response letter to the corporation addressing the NDA. The FDA has given Oleogel-S10 Rare Pediatric Disease Designation, Fast Track, and Priority Review for the dystrophic epidermolysis bullosa treatment. Furthermore, the US FDA and EMA have designated it as an orphan drug.

The EMA has authorized Oleogel-S10 (Filsuvez) for the treatment of DEB cutaneous symptoms. The company is currently in Phase III testing to investigate the effectiveness and safety of Oleogel-S10 in individuals with hereditary EB.

Beremagene geperpavec (KB103) (Krystal Biotech) is an investigational non-invasive, topical, redosable gene therapy that delivers two copies of the COL7A1 gene when administered directly to DEB wounds. The drug is currently being studied in Phase II and Phase III clinical studies to see if topical B-vec may safely and effectively improve healing of DEB patient wounds and measure change from baseline in global investigator evaluations and patient-reported outcomes.

The FDA and EMA have given B-VEC Orphan Drug Designation for dystrophic epidermolysis bullosa treatment, and the FDA has granted B-VEC Fast Track designation and Rare Pediatric Designation for dystrophic epidermolysis bullosa treatment. Furthermore, the FDA also granted B-VEC Regenerative Medicine Advanced Therapy (RMAT), and the EMA granted PRIority MEdicines ("PRIME") for B-VEC to treat DEB.

EB-101 (Abeona Therapeutics) is an RDEB cell treatment that is autologous and gene-corrected. The company is currently undertaking a Phase III trial to assess the safety and efficacy of the surgical application of EB-101 as a therapy for RDEB. The US FDA and EMA have designated EB-101 as an orphan drug for dystrophic epidermolysis bullosa treatment. Furthermore, the US FDA has granted the drug Fast Track Designation, Breakthrough Therapy Designation, and Regenerative Medicine Advanced Therapy Designation.

PTR-01 (BridgeBio) is an investigational protein replacement therapy for the treatment of RDEB that employs recombinant collagen type VII (Rc7). The US FDA and EMA designated PTR-01 as an orphan drug to aid in the development of recessive dystrophic epidermolysis bullosa treatments. In addition, the FDA granted the drug FastTrack Designation to expedite its development and evaluation. This dystrophic epidermolysis bullosa drug is currently being studied in Phase II clinical studies for RDEB.

Expected Roadblocks

There are certain factors that are hindering the growth of the dystrophic epidermolysis bullosa treatment market. The cost of improving QOL and wound care is sometimes expensive and may not be reimbursed by healthcare insurers. Additionally, many countries do not have a patient registry system for epidermolysis bullosa. Despite ongoing advances in gene and cell dystrophic epidermolysis bullosa treatments, a lack of understanding about the disease may limit the expansion of the DEB care and dystrophic epidermolysis bullosa treatment market. Moreover, gene therapy is expensive; also, the healthcare system in most nations, including the United States, is not designed to handle huge one-time expenditures.

However, as there is no disease-specific authorized dystrophic epidermolysis bullosa treatment, developing therapies offer a major possibility. The robust clinical dystrophic epidermolysis bullosa pipeline containing emerging drugs with novel RoA and MoA holds promising future dystrophic epidermolysis bullosa therapies. Moreover, patients willingness to pay for costly treatments such as gene therapy, cell-based therapy, and molecular therapy to treat the disease, thus boosting the dystrophic epidermolysis bullosa treatment market. Additionally, the widespread dystrophic epidermolysis bullosa prevalence is also a prominent factor bolstering the dystrophic epidermolysis bullosa treatment market. 

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