Date: 2018-08-07
Type of information: Granting of the orphan status in the US
Product name: KB105
Compound: replication-incompetent, non-integrating Herpes simplex type 1 (HSV-1) vector expressing the human transglutaminase-1 (TGM-1) enzyme
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism:
- gene therapy. KB105 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s STAR-D platform to deliver functional human TGM-1 gene directly to the patients’ dividing and non-dividing skin cells.
- Transglutaminase 1 (TGM-1) is an essential epidermal enzyme that facilitates the formation of the epidermal barrier, which prevents dehydration, and protects the skin from unwanted toxins and surface microorganisms. The loss of TGM-1-activity results in the severe genetic skin disease autosomal recessive congenital ichthyosis (ARCI). Most patients with a TGM-1-deficiency exhibit life-long pronounced scaling with increased transepidermal water loss (TEWL). The scales are plate-like, often of a dark color, and cover the whole body surface area. Erythroderma is either absent or minimal. Such patients usually have ectropion and, at times, eclabium, hypoplasia of joint and nasal cartilage, scarring alopecia, especially at the edge of the scalp, and palmoplantar keratoderma. Additional complications include episodes of sepsis, fluid and electrolyte imbalances due to impaired skin barrier function, and failure to thrive, especially during neonatal period and infancy. Severe heat intolerance, and nail dystrophy are also frequently observed. TGM-1-deficient ARCI is associated with increased mortality in the neonatal period and has a dramatic impact on quality of life. No efficient treatment is available; current therapy only relieves some symptoms.
Company: Krystal Biotech (USA - PA)
Disease: TGM-1-deficient autosomal recessive congenital ichthyosis (ARCI)
Latest news:
- • On August 23, 2018, Krystal Biotech announced that the FDA has granted a Rare Pediatric Disease Designation (RPDD) to KB105, for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis (ARCI).
- • On August 7, 2018, the FDA has granted orphan drug designation to Krystal Biotech’s second product candidate, KB105, currently in preclinical development for treatment of patients with transglutaminase 1 (TGM-1) deficient autosomal recessive congenital ichthyosis .
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2018-08-07
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
