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• The FDA approved the oral drug nitisinone, to be marketed under the brand name Harliku, for use in adult patients with alkaptonuria.

• Affecting one in 100,000 to 250,000 people worldwide, alkaptonuria (AKU) is an ultra-rare genetic disease caused by a deficiency in the HGD an enzyme. Patients with AKU experience a pathological build-up of a particular acid in the skin, leading to ochronosis, or the bluish or reddish discoloration of patches of skin.

• A small-molecule drug, Harliku works by blocking a key enzyme in the HGD cascade, ultimately reducing urine HGA levels. Data from a 40-patient randomized clinical trial showed that the drug improved pain, physical function and energy after three years of treatment. (BioSpace)

• Dupixent (dupilumab) gained another approved indication, as the first targeted drug for bullous pemphigoid, a rare blistering skin disease that largely affects elderly patients.

• Approval of the injectable interleukin (IL)-4 receptor alpha antagonist was supported by data on 106 adults with moderate-to-severe bullous pemphigoid from the phase II/III ADEPT trial, which tested dupilumab (300 mg) versus placebo atop of standard oral corticosteroids.

• Roughly 27,000 U.S. adults have bullous pemphigoid that is uncontrolled by systemic corticosteroids, according to the drugmakers.

• The chronic and relapsing condition, which is associated with type 2 inflammation, is characterized by intense itching and reddening of the skin. (Medpage Today)

• The FDA approved the TROP2-targeting drug Datroway in a new lung cancer indication, broadening the amount of patients for the companies’ hopeful next big blockbuster.

• Datroway is now indicated to treat EGFR-mutated, locally advanced or metastatic non-small cell lung cancer. The FDA’s accelerated approval decision came nearly three weeks ahead of the July 12 PDUFA date.

• AstraZeneca will need to confirm the drug’s benefit in an additional phase 3 study called TROPION-Lung15, a spokesperson said. A readout is expected in June 2026. (Endpoints)

• The monoclonal antibody, given to babies under 8 months of age entering their first RSV season, is the second product of this kind to enter the market, joining Sanofi and AstraZeneca’s Beyfortus in the RSV toolkit.

• Enflonsia was approved by the FDA in early June. At the time, company officials said Merck planned to start taking orders for the product in July and would deliver the doses in the US this fall, in advance of the start of the RSV season.

• There is currently no CDC director or even an acting director.

• Whether health secretary Robert F. Kennedy Jr. will endorse the recommendation remains to be seen.

• There were six recommendations that passed at the ACIP’s last meeting, in mid-April. Kennedy approved three, leaving another three — related to a new meningococcal vaccine and expanded use of RSV vaccines for adults — in limbo.

• Meissner, who teaches medicine at Dartmouth’s Geisel School of Medicine, tried repeatedly to impress upon his fellow members the need for tools to help lower the burden of RSV infections, the leading cause of hospitalization of children in the U.S.

“These are truly remarkable products. They are safe and they are effective,” Meissner insisted.

• Most of the replacements he has named to date bring little experience on vaccination issues to the committee, a point which has been evident multiple times throughout the meeting, which began yesterday.

• Eli Lilly’s tirzepatide, marketed as Mounjaro for diabetes and Zepbound for weight loss, is expected to rake in $62 billion a year by 2030, according to Evaluate.

• This class of drugs is expected to have five of the top 10 slots on the best-sellers list by 2030 and will be four of the most promising candidates in the pipeline.

• At least, that’s what his gravestone says at the beginning of “Friedreich’s Back,” a marketing campaign for the drug Skyclarys, Biogen’s treatment for the ultra-rare genetic condition, Friedreich ataxia.

• But when Friedreich’s corpse reaches a hand up out of the ground, zombie-movie style, the ad takes a turn.

• After just three months, the videos hit 10 million views on Meta and TikTok, which is roughly 600 times the size of the entire patient population.

• It also drove more than 5,000 conversions to the Skyclarys website during that time, which is astonishing considering the total US patient population is just 5,000.

• And the campaign took home the Gold Lion award at this year’s Cannes Lion Festival of Creativity in France.