Regulatory channel feed

Trel­e­gy gets FDA thumbs-up for asth­ma, be­com­ing first triple-com­bo ther­a­py in the US

One of GSK’s biggest blockbusters just got a whole new indication to play with.

Trelegy Ellipta received the green light from the FDA late Wednesday for adults with asthma, becoming the first triple-therapy combination drug to treat the condition in the United States. The medicine had been approved for use in chronic obstructive pulmonary disease since 2017 and functions as a once-daily inhalation.

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Doug Ingram, Sarepta CEO (Sarepta)

FDA knocks back Sarep­ta in Duchenne gene ther­a­py race with Pfiz­er, but an­a­lysts urge cau­tion

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positive results and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a “program update” for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but “needed additional dialogue” with the agency to confirm.

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Levi Garraway, Roche CMO

Roche nabs an FDA OK for Gavre­to, trig­ger­ing a heavy­weight bout with ri­vals at Eli Lil­ly

Four months after Eli Lilly’s Retevmo got the first FDA OK to treat RET-altered tumors, Roche and Blueprint Medicines are stepping into the ring with recently-approved Gavreto.

The Roche drug was approved on Friday to treat non-small cell lung cancer (NSCLC) patients with RET fusions, which are only present in about 1% to 2% of patients, according to Roche CMO Levi Garraway, who called the OK  “an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease.”

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Michelle McMurry-Heath, BIO CEO (BIO)

BIO and a string of CEOs is­sue a call for FDA in­de­pen­dence and against sci­ence-by-press-re­lease

It appears to be open letter week in biotech.

On Sunday, Scripps Research’s Eric Topol penned a scathing letter in Medscape calling for FDA commissioner Stephen Hahn to resign after a series of alleged missteps and political concessions.  And today, in a carefully-worded piece, the head of BIO and eight well-known biotech CEOs issued a call for the FDA’s independence and for biotechs — with the world spotlight on them — to be on their best behavior.

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FDA sends warn­ing let­ter to My­lan over 'i­nad­e­quate' man­u­fac­tur­ing process­es at In­dia plant

Mylan has once again run afoul of the FDA over its lack of upkeep at manufacturing plants.

For the second time in the last 12 months, the agency has sent a warning letter to the generic drugmaker calling out its failure to maintain industry-standard cleaning procedures that prevent contamination. In the letter, sent on Aug. 20 and published Tuesday, regulators also allege Mylan $MYL did not test incoming API for impurities, have mechanisms in place to detect such impurities from entering products, or keep cleaning records for bulk storage tanks.

Bris­tol My­ers snares an­oth­er ap­proval from the old Cel­gene pipeline, nab­bing an over­looked OK

Bristol Myers Squibb has gotten another approval from the old Celgene pipeline yesterday, this time for a once low-profile compound.

The FDA green-lighted Onureg, an oral drug previously known as CC-486. It’s chemically known as azacitidine — the same compound that in its IV form, branded as Vidaza, has long been used to treat acute myeloid leukemia and myelodysplastic syndromes. For Bristol Myers, it marks the second major approval since the Celgene merger was completed, after the multiple sclerosis drug Zeposia was OK’d earlier this year.

FDA re­leas­es PDU­FA VI pro­gram re­port on com­bi­na­tion prod­ucts

A new independent report on the FDA’s review practices for combination products under the Prescription Drug User Fee Act (PDUFA VI) gives the agency generally positive marks but finds room for refinements in communication and technology to improve the pre-submission and review process.

The report, commissioned by FDA as part of its PDUFA VI commitments, was conducted by Eastern Research Group and looked at FDA staff and sponsor experiences with combination products reviews during the pre-request for designation (pre-RFD), RFD, inter-center consult request (ICCR) and application submission stages.

New analy­sis shows in­clisir­an con­sis­ten­cy in LDL-C re­duc­tion as No­var­tis read­ies for FDA de­ci­sion

As the FDA continues its review of the former-The Medicines Company, now-Novartis candidate inclisiran ahead of an expected PDUFA before the year is out, the Swiss pharma released pooled data that it hopes bolsters the case for approval.

A post-hoc analysis of two Phase III inclisiran trials showed consistency in efficacy and safety among patients with both hyperlipidemia and atherosclerotic cardiovascular disease despite statin therapy, with 99% of patients showing greater than 30% placebo-adjusted reduction in LDL-C levels. The average reduction was 54.1% from baseline in the 2,300 individuals between the two studies.

FDA drafts rec­om­men­da­tions for study­ing can­cer drugs in pa­tients with CNS metas­tases

The FDA on Wednesday issued draft guidance providing recommendations to drugmakers on evaluating cancer drugs in patients with central nervous system (CNS) metastases. FDA says the guidance is meant to inform clinical trial designs to support product labeling that describes antitumor activity of drugs or biologics in patients with CNS metastases from solid tumors originating in other parts of the body.

Cos­mo spin­out Cas­sio­pea wins FDA ap­proval for new ac­ne drug

A small Swiss-listed biotech spinout secured FDA approval for their first drug, setting them up to compete in a crowded market for acne.

Cassiopea received an okay for Winlevi, the drug that has been at the top of the company’s pipeline since the company spun out from the Swiss pharma Cosmo in 2015. It will take a little bit longer to market, though, likely because the company has little commercial infrastructure so far.  The biotech said they plan to start early 2021. A price will be announced in Q4, Reuters reported.