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Shares of BridgeBio Pharma jumped 6% in Monday’s pre-market session after the US Food and Drug Administration (FDA) approved its Nulibry therapy for the treatment of Molybdenum cofactor deficiency (MoCD) Type A. MoCD is a genetic disorder that causes non-reversible neurological damage and has high death rate among infants.
BridgeBio Pharma (BBIO) and its affiliate Origin Biosciences said that the the approval was granted following Phase 3 clinical trial data, which demonstrated efficacy of the treatment in comparison to a natural history study. MoCD Type A is an ultrarare disease affecting less than 150 patients globally. It occurs after birth and the median survival rate is 4 years.
Notably, the treatment received an orphan drug designation, breakthrough therapy designation as well as rare pediatric disease designation from the FDA.
BridgeBio Pharma CEO Neil Kumar said, “The FDA’s approval of Nulibry means that patients with MoCD Type A and their families have an approved therapy for the first time. It also reflects our belief that every life matters and that no disease is too rare to address.”
BridgeBio has more than 30 products in its development pipeline at different stages. (See BridgeBio stock analysis on TipRanks)
Following BridgeBio’s recent earnings release, Raymond James analyst Dane Leone raised the stock’s price target to $84 (18.9% upside potential) from $50 and maintained a Buy rating. In a note to investors, the analyst said, “We are raising our price target based upon our 2021 outlook for clinical catalysts and readouts across the BridgBio portfolio.”
From the rest of Wall Street analysts, BridgeBio scores a Strong Buy consensus rating based on 8 Buys and 1 Hold. The average analyst price target of $74.44 implies about 5.3% upside from current levels. That’s after shares propelled 119% over the past year.